Cell (biology) – Wikipedia

The cell (from Latin cella, meaning "small room"[1]) is the basic structural, functional, and biological unit of all known living organisms. A cell is the smallest unit of life that can replicate independently, and cells are often called the "building blocks of life". The study of cells is called cell biology.

Cells consist of cytoplasm enclosed within a membrane, which contains many biomolecules such as proteins and nucleic acids.[2] Organisms can be classified as unicellular (consisting of a single cell; including bacteria) or multicellular (including plants and animals). While the number of cells in plants and animals varies from species to species, humans contain more than 10 trillion (1012) cells. Most plant and animal cells are visible only under a microscope, with dimensions between 1 and 100micrometres.[4]

The cell was discovered by Robert Hooke in 1665, who named the biological unit for its resemblance to cells inhabited by Christian monks in a monastery.[5][6]Cell theory, first developed in 1839 by Matthias Jakob Schleiden and Theodor Schwann, states that all organisms are composed of one or more cells, that cells are the fundamental unit of structure and function in all living organisms, that all cells come from preexisting cells, and that all cells contain the hereditary information necessary for regulating cell functions and for transmitting information to the next generation of cells.[7] Cells emerged on Earth at least 3.5 billion years ago.[8][9][10]

Cells are of two types, eukaryotic, which contain a nucleus, and prokaryotic, which do not. Prokaryotes are single-celled organisms, while eukaryotes can be either single-celled or multicellular.

Prokaryotic cells were the first form of life on Earth, characterised by having vital biological processes including cell signaling and being self-sustaining. They are simpler and smaller than eukaryotic cells, and lack membrane-bound organelles such as the nucleus. Prokaryotes include two of the domains of life, bacteria and archaea. The DNA of a prokaryotic cell consists of a single chromosome that is in direct contact with the cytoplasm. The nuclear region in the cytoplasm is called the nucleoid. Most prokaryotes are the smallest of all organisms ranging from 0.5 to 2.0m in diameter.[12]

A prokaryotic cell has three architectural regions:

Plants, animals, fungi, slime moulds, protozoa, and algae are all eukaryotic. These cells are about fifteen times wider than a typical prokaryote and can be as much as a thousand times greater in volume. The main distinguishing feature of eukaryotes as compared to prokaryotes is compartmentalization: the presence of membrane-bound organelles (compartments) in which specific metabolic activities take place. Most important among these is a cell nucleus, an organelle that houses the cell's DNA. This nucleus gives the eukaryote its name, which means "true kernel (nucleus)". Other differences include:

All cells, whether prokaryotic or eukaryotic, have a membrane that envelops the cell, regulates what moves in and out (selectively permeable), and maintains the electric potential of the cell. Inside the membrane, the cytoplasm takes up most of the cell's volume. All cells (except red blood cells which lack a cell nucleus and most organelles to accommodate maximum space for hemoglobin) possess DNA, the hereditary material of genes, and RNA, containing the information necessary to build various proteins such as enzymes, the cell's primary machinery. There are also other kinds of biomolecules in cells. This article lists these primary components of the cell, then briefly describes their function.

The cell membrane, or plasma membrane, is a biological membrane that surrounds the cytoplasm of a cell. In animals, the plasma membrane is the outer boundary of the cell, while in plants and prokaryotes it is usually covered by a cell wall. This membrane serves to separate and protect a cell from its surrounding environment and is made mostly from a double layer of phospholipids, which are amphiphilic (partly hydrophobic and partly hydrophilic). Hence, the layer is called a phospholipid bilayer, or sometimes a fluid mosaic membrane. Embedded within this membrane is a variety of protein molecules that act as channels and pumps that move different molecules into and out of the cell. The membrane is said to be 'semi-permeable', in that it can either let a substance (molecule or ion) pass through freely, pass through to a limited extent or not pass through at all. Cell surface membranes also contain receptor proteins that allow cells to detect external signaling molecules such as hormones.

The cytoskeleton acts to organize and maintain the cell's shape; anchors organelles in place; helps during endocytosis, the uptake of external materials by a cell, and cytokinesis, the separation of daughter cells after cell division; and moves parts of the cell in processes of growth and mobility. The eukaryotic cytoskeleton is composed of microfilaments, intermediate filaments and microtubules. There are a great number of proteins associated with them, each controlling a cell's structure by directing, bundling, and aligning filaments. The prokaryotic cytoskeleton is less well-studied but is involved in the maintenance of cell shape, polarity and cytokinesis.[17] The subunit protein of microfilaments is a small, monomeric protein called actin. The subunit of microtubules is a dimeric molecule called tubulin. Intermediate filaments are heteropolymers whose subunits vary among the cell types in different tissues. But some of the subunit protein of intermediate filaments include vimentin, desmin, lamin (lamins A, B and C), keratin (multiple acidic and basic keratins), neurofilament proteins (NF - L, NF - M).

Two different kinds of genetic material exist: deoxyribonucleic acid (DNA) and ribonucleic acid (RNA). Cells use DNA for their long-term information storage. The biological information contained in an organism is encoded in its DNA sequence. RNA is used for information transport (e.g., mRNA) and enzymatic functions (e.g., ribosomal RNA). Transfer RNA (tRNA) molecules are used to add amino acids during protein translation.

Prokaryotic genetic material is organized in a simple circular DNA molecule (the bacterial chromosome) in the nucleoid region of the cytoplasm. Eukaryotic genetic material is divided into different, linear molecules called chromosomes inside a discrete nucleus, usually with additional genetic material in some organelles like mitochondria and chloroplasts (see endosymbiotic theory).

A human cell has genetic material contained in the cell nucleus (the nuclear genome) and in the mitochondria (the mitochondrial genome). In humans the nuclear genome is divided into 46 linear DNA molecules called chromosomes, including 22 homologous chromosome pairs and a pair of sex chromosomes. The mitochondrial genome is a circular DNA molecule distinct from the nuclear DNA. Although the mitochondrial DNA is very small compared to nuclear chromosomes, it codes for 13 proteins involved in mitochondrial energy production and specific tRNAs.

Foreign genetic material (most commonly DNA) can also be artificially introduced into the cell by a process called transfection. This can be transient, if the DNA is not inserted into the cell's genome, or stable, if it is. Certain viruses also insert their genetic material into the genome.

Organelles are parts of the cell which are adapted and/or specialized for carrying out one or more vital functions, analogous to the organs of the human body (such as the heart, lung, and kidney, with each organ performing a different function). Both eukaryotic and prokaryotic cells have organelles, but prokaryotic organelles are generally simpler and are not membrane-bound.

There are several types of organelles in a cell. Some (such as the nucleus and golgi apparatus) are typically solitary, while others (such as mitochondria, chloroplasts, peroxisomes and lysosomes) can be numerous (hundreds to thousands). The cytosol is the gelatinous fluid that fills the cell and surrounds the organelles.

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Many cells also have structures which exist wholly or partially outside the cell membrane. These structures are notable because they are not protected from the external environment by the semipermeable cell membrane. In order to assemble these structures, their components must be carried across the cell membrane by export processes.

Many types of prokaryotic and eukaryotic cells have a cell wall. The cell wall acts to protect the cell mechanically and chemically from its environment, and is an additional layer of protection to the cell membrane. Different types of cell have cell walls made up of different materials; plant cell walls are primarily made up of cellulose, fungi cell walls are made up of chitin and bacteria cell walls are made up of peptidoglycan.

A gelatinous capsule is present in some bacteria outside the cell membrane and cell wall. The capsule may be polysaccharide as in pneumococci, meningococci or polypeptide as Bacillus anthracis or hyaluronic acid as in streptococci. Capsules are not marked by normal staining protocols and can be detected by India ink or methyl blue; which allows for higher contrast between the cells for observation.[19]:87

Flagella are organelles for cellular mobility. The bacterial flagellum stretches from cytoplasm through the cell membrane(s) and extrudes through the cell wall. They are long and thick thread-like appendages, protein in nature. A different type of flagellum is found in archaea and a different type is found in eukaryotes.

A fimbria also known as a pilus is a short, thin, hair-like filament found on the surface of bacteria. Fimbriae, or pili are formed of a protein called pilin (antigenic) and are responsible for attachment of bacteria to specific receptors of human cell (cell adhesion). There are special types of specific pili involved in bacterial conjugation.

Between successive cell divisions, cells grow through the functioning of cellular metabolism. Cell metabolism is the process by which individual cells process nutrient molecules. Metabolism has two distinct divisions: catabolism, in which the cell breaks down complex molecules to produce energy and reducing power, and anabolism, in which the cell uses energy and reducing power to construct complex molecules and perform other biological functions. Complex sugars consumed by the organism can be broken down into simpler sugar molecules called monosaccharides such as glucose. Once inside the cell, glucose is broken down to make adenosine triphosphate (ATP), a molecule that possesses readily available energy, through two different pathways.

Cell division involves a single cell (called a mother cell) dividing into two daughter cells. This leads to growth in multicellular organisms (the growth of tissue) and to procreation (vegetative reproduction) in unicellular organisms. Prokaryotic cells divide by binary fission, while eukaryotic cells usually undergo a process of nuclear division, called mitosis, followed by division of the cell, called cytokinesis. A diploid cell may also undergo meiosis to produce haploid cells, usually four. Haploid cells serve as gametes in multicellular organisms, fusing to form new diploid cells.

DNA replication, or the process of duplicating a cell's genome, always happens when a cell divides through mitosis or binary fission. This occurs during the S phase of the cell cycle.

In meiosis, the DNA is replicated only once, while the cell divides twice. DNA replication only occurs before meiosis I. DNA replication does not occur when the cells divide the second time, in meiosis II.[20] Replication, like all cellular activities, requires specialized proteins for carrying out the job.

Cells are capable of synthesizing new proteins, which are essential for the modulation and maintenance of cellular activities. This process involves the formation of new protein molecules from amino acid building blocks based on information encoded in DNA/RNA. Protein synthesis generally consists of two major steps: transcription and translation.

Transcription is the process where genetic information in DNA is used to produce a complementary RNA strand. This RNA strand is then processed to give messenger RNA (mRNA), which is free to migrate through the cell. mRNA molecules bind to protein-RNA complexes called ribosomes located in the cytosol, where they are translated into polypeptide sequences. The ribosome mediates the formation of a polypeptide sequence based on the mRNA sequence. The mRNA sequence directly relates to the polypeptide sequence by binding to transfer RNA (tRNA) adapter molecules in binding pockets within the ribosome. The new polypeptide then folds into a functional three-dimensional protein molecule.

Unicellular organisms can move in order to find food or escape predators. Common mechanisms of motion include flagella and cilia.

In multicellular organisms, cells can move during processes such as wound healing, the immune response and cancer metastasis. For example, in wound healing in animals, white blood cells move to the wound site to kill the microorganisms that cause infection. Cell motility involves many receptors, crosslinking, bundling, binding, adhesion, motor and other proteins.[21] The process is divided into three steps protrusion of the leading edge of the cell, adhesion of the leading edge and de-adhesion at the cell body and rear, and cytoskeletal contraction to pull the cell forward. Each step is driven by physical forces generated by unique segments of the cytoskeleton.[22][23]

Multicellular organisms are organisms that consist of more than one cell, in contrast to single-celled organisms.[24]

In complex multicellular organisms, cells specialize into different cell types that are adapted to particular functions. In mammals, major cell types include skin cells, muscle cells, neurons, blood cells, fibroblasts, stem cells, and others. Cell types differ both in appearance and function, yet are genetically identical. Cells are able to be of the same genotype but of different cell type due to the differential expression of the genes they contain.

Most distinct cell types arise from a single totipotent cell, called a zygote, that differentiates into hundreds of different cell types during the course of development. Differentiation of cells is driven by different environmental cues (such as cellcell interaction) and intrinsic differences (such as those caused by the uneven distribution of molecules during division).

Multicellularity has evolved independently at least 25 times,[25] including in some prokaryotes, like cyanobacteria, myxobacteria, actinomycetes, Magnetoglobus multicellularis or Methanosarcina. However, complex multicellular organisms evolved only in six eukaryotic groups: animals, fungi, brown algae, red algae, green algae, and plants.[26] It evolved repeatedly for plants (Chloroplastida), once or twice for animals, once for brown algae, and perhaps several times for fungi, slime molds, and red algae.[27] Multicellularity may have evolved from colonies of interdependent organisms, from cellularization, or from organisms in symbiotic relationships.

The first evidence of multicellularity is from cyanobacteria-like organisms that lived between 3 and 3.5 billion years ago.[25] Other early fossils of multicellular organisms include the contested Grypania spiralis and the fossils of the black shales of the Palaeoproterozoic Francevillian Group Fossil B Formation in Gabon.[28]

The evolution of multicellularity from unicellular ancestors has been replicated in the laboratory, in evolution experiments using predation as the selective pressure.[25]

The origin of cells has to do with the origin of life, which began the history of life on Earth.

There are several theories about the origin of small molecules that led to life on the early Earth. They may have been carried to Earth on meteorites (see Murchison meteorite), created at deep-sea vents, or synthesized by lightning in a reducing atmosphere (see MillerUrey experiment). There is little experimental data defining what the first self-replicating forms were. RNA is thought to be the earliest self-replicating molecule, as it is capable of both storing genetic information and catalyzing chemical reactions (see RNA world hypothesis), but some other entity with the potential to self-replicate could have preceded RNA, such as clay or peptide nucleic acid.[29]

Cells emerged at least 3.5 billion years ago.[8][9][10] The current belief is that these cells were heterotrophs. The early cell membranes were probably more simple and permeable than modern ones, with only a single fatty acid chain per lipid. Lipids are known to spontaneously form bilayered vesicles in water, and could have preceded RNA, but the first cell membranes could also have been produced by catalytic RNA, or even have required structural proteins before they could form.[30]

The eukaryotic cell seems to have evolved from a symbiotic community of prokaryotic cells. DNA-bearing organelles like the mitochondria and the chloroplasts are descended from ancient symbiotic oxygen-breathing proteobacteria and cyanobacteria, respectively, which were endosymbiosed by an ancestral archaean prokaryote.

There is still considerable debate about whether organelles like the hydrogenosome predated the origin of mitochondria, or vice versa: see the hydrogen hypothesis for the origin of eukaryotic cells.

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Cell (biology) - Wikipedia

First Clinic-Ready Stem Cell Repository – The Scientist


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First Clinic-Ready Stem Cell Repository
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WELLCOME IMAGES, ANNIE CAVANAGHIn an effort to make it easier for researchers to develop stem cellbased therapies, the UK Stem Cell Bank is offering several ready-to-go human embryonic stem cell (hESC) lines derived from donated tissue that ...
Can Stem Cell 'Patch' Help Heart Failure?WebMD
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First Clinic-Ready Stem Cell Repository - The Scientist

Human Embryonic Stem Cells (hESC) Market to Reach $1 Billion by 2025 – Application of hESCs as a Promising Donor … – Yahoo Finance

DUBLIN--(BUSINESS WIRE)--

Research and Markets has announced the addition of the "Human Embryonic Stem Cells (hESC) Market, 2014 - 2025" report to their offering.

The global human embryonic stem cells (hESCs) market is anticipated to reach USD 1.06 billion by 2025. Application of hESCs as a promising donor source for cellular transplantation therapies is anticipated to bolster progress through to 2025. hESCs technology tends to be useful for tissue engineering in humans due to high histocompatibility between host and graft.

Maintenance of developmental potential for contribution of derivatives of all three germ layers is an important feature of these cells. This ability remains consistent even after clonal derivation or prolonged undifferentiated proliferation, thus pronouncing its accelerated uptake.

In addition, these are capable in expressing high level of alkaline phosphatase, key transcription factors, and telomerase. These factors are found to be of great importance in the maintenance of the inner cellular mass pluripotency.

Furthermore, hESCs can be easily differentiated into defined neurons, neural lineages, oligodendrocytes, and astrocytes. Aforementioned characteristic makes it useful in studying the sequence of events that take place during early neurodevelopment.

However, use of stem cells derived from viable embryos is fraught with ethical issues, prompting scientists to explore other methods to generate ESCs. The other methods include derivation of embryonic germ cells, stem cells from dead embryos, and other techniques.

Companies Mentioned

Key Topics Covered:

1 Research Methodology

2 Executive Summary

3 Human Embryonic Stem Cells Market Variables, Trends & Scope

4 Human Embryonic Stem Cells Market: Application Estimates & Trend Analysis

5 Human Embryonic Stem Cells Market: Regional Estimates & Trend Analysis, by Application

6 Competitive Landscape

For more information about this report visit http://www.researchandmarkets.com/research/w7n75n/human_embryonic

View source version on businesswire.com: http://www.businesswire.com/news/home/20170407005275/en/

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Human Embryonic Stem Cells (hESC) Market to Reach $1 Billion by 2025 - Application of hESCs as a Promising Donor ... - Yahoo Finance

Stem cells: Single-use bioreactors and microcarriers can overcome scale-up issues, experts – BioPharma-Reporter.com

The only practical way to scale-up volumes of mesenchymal stem cells (MSCs) is by using microcarriers in single-use bioreactors, say scientists from A*STAR and Instituto Superior Tcnico.

MSCs are multipotent stromal cells that can differentiate into a variety of cell types which are being investigated for tissue engineering and cellular therapies.

Such cells come from bone marrow, adipose tissue and umbilical cord blood but are very rare, according to Ana Fernandes-Platzgummer, a research scientist for the Stem Cell Engineering Research Group at the Instituto Superior Tcnico in Lisbon, Portugal.

Totipotent cells can form all the cell types in a body, plus the extraembryonic, or placental, cells. The only totipotent cells are embryonic cells within the first couple of cell divisions after fertilisation.

Pluripotent cells can give rise to all of the cell types that make up the body. While embryonic stem cells are considered pluripotent, this class includes induced pluripotent stem cells (iPSC) derived from skin or blood cells that have been reprogrammed back into an embryonic-like pluripotent state.

Multipotent cells are more limited than pluripotent cells but can develop into more than one cell type. This class includes mesenchymal stem cells (MSCs) derived from bone marrow, adipose tissue and umbilical cord blood, and hematopoietic stem cells (HSCs) derived from mesoderm and located in the red bone marrow.

There are only about 100,000 stem cells in an umbilical cord, she told delegates at the 1st Stem Cell Community day in Germany this week. For cellular therapies we need doses of more than one million cells per kg [ideal (IBW) or actual (ABW) body weight] so we need to expand these cells.

Scale-up challenges

Stem cells can be successfully cultivated using flasks and labscale-volume bioreactors but there are many problems in monitoring and controlling growth, and issues with productivity and cell harvest. Therefore scale-up is a problem, hindered further due to a lack of technologies and processes available to cell therapy makers.

The event in Hamburg organised by bioprocessing tech firm Eppendorf looked to address these challenges in stem cell cultivation and scale-up by bringing together industry and academia.

And Fernandes-Platzgummer said that research by the Instituto Superior Tcnico together with Thermo Fisher-owned Life Technologies showed positive results in the expansion of human MSCs from different sources using a fully-controlled stirred-tank bioreactor combined with microcarrier technology.

The advantage of this is its easy scalability, the high surface area [of the microcarrier], the ability to monitor and control cultivation, and the reduced labour costs and risks of contamination, she said.

After five days cultivation the team produced clinically-relevant cell numbers, she added, using an 800ml spinner flask bioreactor, Thermo Fishers serum-free medium StemPro and reagent TrypLE Select CTS, and plastic microcarriers coated with the xeno-free substrate CELLstart (also made by Thermo Fisher).

'10,000 doses per year, each of a billion cells'

In a separate presentation, Steve Oh principal scientist and associate director at the Bioprocessing Technology Institute (BTI), part of Singapores Agency for Science, Technology and Research (A*STAR) said a similar set-up had shown promise in moving MSC cultivation into scalable technologies and his team is trying to move to a 15L scale.

However, the goal for MSC-based therapies would be producing commercial volumes of 10,000 doses per year, each of a billion cells from the onset, he added.

We looked at all the approaches and really the only practical experience I have of a technology that will succeed is microcarrier technology using single-use bioreactors, he said.

Oh added microcarriers produce higher cell densities with the same amount of media while allowing greater control of the process by providing another metric to configure.

Furthermore, having only thin layers of cells between each carrier offers benefits in the harvesting of stem cells which he said is as problematic as cultivation due to the large aggregates of cell clusters formed which are difficult to break up.

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Stem cells: Single-use bioreactors and microcarriers can overcome scale-up issues, experts - BioPharma-Reporter.com

Nerve cells actively repress alternative cell fates | News Center … – Stanford Medical Center Report

Until now, researchers have focused only on identifying these types of single-lineage repressors, said Wernig. The concept of an everything but repressor is entirely new.

In 2010, Wernig showed that it is possible to convert skin cells into functional neurons over the course of three weeks by exposing them to a combination of just three proteins that are typically expressed in neurons. This direct reprogramming bypassed a step called induced pluripotency that many scientists had thought was necessary to transform one cell type into another.

One of the proteins necessary to accomplish the transformation of skin to neurons was Myt1l. But until this study the researchers were unaware precisely how it functioned.

Usually we think in terms about what regulatory programs need to be activated to direct a cell to a specific developmental state, said Wernig. So we were surprised when we took a closer look and saw that Myt1l was actually suppressing the expression of many genes.

These genes, the researchers found, encoded proteins important for the development of lung, heart, liver, cartilage and other types of non-neuronal tissue. Furthermore, two of the proteins, Notch and Wnt, are known to actively block neurogenesis in the developing brain.

Blocking Myt1l expression in the brains of embryonic mice reduced the number of mature neurons that developed in the animals. Furthermore, knocking down Myt1l expression in mature neurons caused them to express lower-than-normal levels of neural-specific genes and to fire less readily in response to an electrical pulse.

Wernig and his colleagues contrasted the effect of Myt1l with that of another protein called Ascl1, which is required to directly reprogram skin fibroblasts into neurons. Ascl1 is known to specifically induce the expression of neuronal genes in the fibroblasts.

Together, these proteins work as a perfect team to funnel a developing cell, or a cell that is being reprogrammed, into the desired cell fate, said Wernig. Its a beautiful scenario that both blocks the fibroblast program and promotes the neuronal program. My gut feeling would be that there are many more master repressors like Myt1l to be found for specific cell types, each of which would block all but one cell fate.

Wernig is a member of Stanfords Cardiovascular Institute, Child Health Research Institute, Cancer Institute, Neurosciences Institute and Bio-X.

Other Stanford co-authors of the paper are postdoctoral scholars Soham Chanda, PhD, Bo Zhou, PhD, Xuecai Ge, PhD, and Philip Brennecke, PhD; graduate students Cheen Ang, Thomas Vierbuchen and Daniel Fuentes; research assistant Sarah Grieder; undergraduate student Brandon Walker; professor of genetics Lars Steinmetz, PhD; and professor of molecular and cellular biology Thomas Sudhof, MD.

The research was supported by the German Research Foundation, the National Institutes of Health (grant R01MH092931), the California Institute for Regenerative Medicine, the New York Stem Cell Foundation, the Howard Hughes Medical Institute, the Swedish Research Council, the Swedish Government Initiative for Strategic Research Institute, the Department of Health and Human Services and Spectrum Child Health.

Stanfords Department of Pathology also supported the work.

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The Alliance for Regenerative Medicine Releases Agenda for Fifth … – Yahoo Finance

WASHINGTON, DC--(Marketwired - Apr 6, 2017) - The Alliance for Regenerative Medicine (ARM) today released the complete agenda for its upcoming fifth annual Cell & Gene Therapy Investor Day, taking place April 27, 2017 in Boston, MA. This event, co-hosted by Piper Jaffray and held in partnership with Cowen and Company is the only investor conference specifically focused on cell and gene therapies, offering exclusive access to the field's most promising companies.

This year's Cell & Gene Therapy Investor Day is expected to attract 350+ attendees, including 175+ active investors and analysts and will feature presentations by 30+ companies, along with panels and fireside chats by the field's foremost thought leaders.

2017 Panel Sessions and Speakers:

Fireside Chat Olivier Danos, Ph.D., Chief Scientific Officer, REGENXBIO (moderator) James M. Wilson, M.D., Ph.D., Rose H. Weiss Professor and Director, Orphan Disease Center; Professor of Medicine and Pediatrics; Director, Gene Therapy Program, Perelman School of Medicine, University of Pennsylvania

Panel I: Cell Therapy Beyond Oncology: Where Does the Greatest Potential Lie? Edward Tenthoff, Managing Director & Senior Research Analyst, Piper Jaffray (moderator) Eduardo Bravo, CEO, TiGenix Adam Gridley, President and CEO, Histogenics Paul Laikind, President and CEO, ViaCyte Chaim Lebovits, President and CEO, BrainStorm Cell Therapeutics Emile Nuwaysir, Ph.D., CEO, BlueRock Therapeutics

Panel II: Gene Therapy: Commercialization Readiness & Market Access Challenges Joshua Schimmer, M.D., Managing Director & Senior Research Analyst, Piper Jaffray (moderator) Faraz Ali, Chief Business Officer, REGENXBIO Sven Kili, M.D., VP and Head of Gene Therapy Development, GlaxoSmithKline Arthur Tzianabos, Ph.D., President and CEO, Homology Medicines Elizabeth White, Ph.D., Assistant VP, Early Commercial Planning, Rare Disease and Gene Therapy, Pfizer Innovative Health

Panel III: Immuno-Oncology: What Are the Key Issues as First Products Approach Commercialization? Timothy Schroeder, CEO, CTI Clinical Trial and Consulting (moderator) Usman Azam, M.D., President and CEO, Tmunity Therapeutics David Epstein, Executive Partner, Flagship Pioneering Rick Fair, President and CEO, Bellicum Pharmaceuticals Jeffrey Walsh, Chief Financial and Strategy Officer, bluebird bio

2017 Presenting Companies:

4D Molecular Therapeutics, Abeona Therapeutics, Adverum Biotechnologies, AGTC, Argos Therapeutics, Audentes Therapeutics, AVROBIO, BioCardia, bluebird bio, Bone Therapeutics, Caladrius Biosciences, Capricor Therapeutics, Celyad, Fate Therapeutics, Fibrocell, GenSight Biologics, Histogenics, Homology Medicines, Juventas Therapeutics, Kiadis Pharma, Lysogene, Mesoblast, MiMedx, Orchard Therapeutics, Oxford BioMedica, Pluristem Therapeutics, Precision Biosciences, ReNeuron, Sangamo Therapeutics, Semma Therapeutics, Synpromics, TiGenix, TxCell, uniQure, Vericel, and Voyager Therapeutics

In addition to the event's co-host Piper Jaffray, sponsors include Cowen and Company; Cognate BioServices; Cryoport; CTI Clinical Trial and Consulting Services; Kawasaki; Lonza; Medpace; PCT, a Caladrius Company; and Edison. For more information please visit the event's website at http://www.arminvestorday.com.

Credentialed investors and life science strategic partners can indicate their interest in attending here. Members of the media interested in attending are asked to please contact Lyndsey Scull at lscull@alliancerm.org.

The event will be held April 27, 2017, beginning at 7:30am at The State Room, 60 State Street, Boston, MA 02109.

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 250 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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The Alliance for Regenerative Medicine Releases Agenda for Fifth ... - Yahoo Finance

Can Stem Cell ‘Patch’ Help Heart Failure? – Montana Standard

WEDNESDAY, April 5, 2017 (HealthDay News) -- Scientists report another step in the use of stem cells to help treat people with debilitating heart failure.

In an early study of 27 patients, Japanese researchers used patients' own muscle stem cells to create a "patch" that was placed on the heart.

Over the next year, the patients generally showed small improvements in their symptoms -- including the ability to walk without becoming breathless and fatigued.

However, experts cautioned that while the results are encouraging, there's a lot of work left ahead before stem cells can be used to treat heart failure.

"They've shown that this approach is feasible," said Dr. Eiran Gorodeski, a heart failure specialist at the Cleveland Clinic in Ohio.

But it's not clear whether the stem-cell tactic was actually effective, said Gorodeski, who was not involved in the study.

That's because the study didn't include a comparison group that did not receive stem cells.

So it's possible, Gorodeski explained, that the "modest" symptom improvements would have happened anyway. All of the patients were on standard medications, and some had heart devices implanted.

Stem cells are primitive cells that mature into the various cells that make up the body's tissues. In the past 15 years or so, scientists have tried to use the cells to help repair some of the damage seen in heart failure.

Heart failure is a progressive disease where the heart muscle is too damaged to efficiently pump blood throughout the body. It often arises after a heart attack.

Symptoms of heart failure include fatigue, breathlessness and swelling in the limbs. The condition cannot be cured, although medications and implantable devices can treat the symptoms.

In the new study, the researchers used stem cells from the patients' own thigh muscle to create a patch they placed on the heart.

That's in contrast to many past studies, where researchers have injected stem cells -- often from patients' bone marrow -- into the heart.

The patch tactic could have some advantages, said senior researcher Dr. Yoshiki Sawa, of Osaka University.

He said animal research suggests that cells in sheet form survive for a longer period, compared to injections.

To test the safety of the approach, Sawa's team recruited 27 patients who had debilitating symptoms despite standard heart failure therapies. The scientists extracted stem cells from each patient's thigh muscle, then cultured the cells so that they formed a sheet.

The sheet was placed on each patient's heart.

The tactic appeared safe, the researchers said, and there were signs of symptom improvements over the next six months to a year.

Why would stem cells from the thigh muscle affect the heart? It's not clear, Sawa acknowledged.

The stem cells don't grow into new heart muscle cells. Instead, Sawa explained, they seem to produce chemicals called cytokines that can promote new blood vessel growth in damaged areas of the heart. The theory, he said, is that "hibernating" cells in the heart muscle can then function better.

Still, it's too soon to know what the new findings mean, said Gorodeski.

This type of trial, called phase 1, is designed to look at the safety and feasibility of a therapy, Gorodeski said. It takes later-phase trials -- where some patients receive the treatment, and others do not -- to prove that a therapy actually works.

Those trials are underway, Sawa said.

Other studies are further along. Last year, researchers reported on a trial testing infusions of stem cells taken from the bone marrow of patients with severe heart failure.

Patients who received the therapy were less likely to die or be hospitalized over the next year, versus those given standard treatment only. But the study was small, and the stem cells had only a minor impact on patients' heart function.

So it's not clear why the stem-cell patients fared better, Gorodeski said.

For now, he stressed, all stem-cell therapies for heart failure remain experimental.

"There's no cell therapy that we can offer patients right now," Gorodeski said.

The message for patients, he added, is that heart failure can be treated, and researchers are looking for "innovative" ways to improve that treatment.

The study was published April 5 in the Journal of the American Heart Association.

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Can Stem Cell 'Patch' Help Heart Failure? - Montana Standard

Global 2017 Cell Therapy Technologies, Markets and Companies … – Yahoo Finance

Dublin, April 06, 2017 (GLOBE NEWSWIRE) -- Research and Markets has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

Cell therapy technologies overlap with those of gene therapy, cancer vaccines, drug delivery, tissue engineering and regenerative medicine. Pharmaceutical applications of stem cells including those in drug discovery are also described. Various types of cells used, methods of preparation and culture, encapsulation and genetic engineering of cells are discussed. Sources of cells, both human and animal (xenotransplantation) are discussed. Methods of delivery of cell therapy range from injections to surgical implantation using special devices.

The cell-based markets was analyzed for 2016, and projected to 2026.The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 305 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells.

Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

Part II: Markets, Companies & Academic Institutions

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit http://www.researchandmarkets.com/research/s5g673/cell_therapy

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Global 2017 Cell Therapy Technologies, Markets and Companies ... - Yahoo Finance