The California Institute for Regenerative Medicine was created in 2004 after 59% of California voters approved California Proposition 71 that governs the allocation of the $3 billion authorized to fund stem cell research in California. The agency was authorized to distribute the money in grants, funded by bonds, over a ten-year period to institutions and scientists throughout California that focused on advancing stem cell research and regenerative medicine. The mission of CIRM is: To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease injury. Wikipedia.
Knoepfler P.S.,University of California at Davis |
Knoepfler P.S.,California Institute for Regenerative Medicine
Advanced Drug Delivery Reviews | Year: 2015
The phrase "bench-to-bedside" is commonly used to describe the translation of basic discoveries such as those on stem cells to the clinic for therapeutic use in human patients. However, there is a key intermediate step in between the bench and the bedside involving governmental regulatory oversight such as by the Food and Drug Administration (FDA) in the United States (US). Thus, it might be more accurate in most cases to describe the stem cell biological drug development process in this way: from bench to FDA to bedside. The intermediate development and regulatory stage for stem cell-based biological drugs is a multifactorial, continually evolving part of the process of developing a biological drug such as a stem cell-based regenerative medicine product. In some situations, stem cell-related products may not be classified as biological drugs in which case the FDA plays a relatively minor role. However, this middle stage is generally a major element of the process and is often colloquially referred to in an ominous way as "The Valley of Death". This moniker seems appropriate because it is at this point, and in particular in the work that ensues after Phase 1, clinical trials that most drug product development is terminated, often due to lack of funding, diseases being refractory to treatment, or regulatory issues. Not surprisingly, workarounds to deal with or entirely avoid this difficult stage of the process are evolving both inside and outside the domains of official regulatory authorities. In some cases these efforts involve the FDA invoking new mechanisms of accelerating the bench to beside process, but in other cases these new pathways bypass the FDA in part or entirely. Together these rapidly changing stem cell product development and regulatory pathways raise many scientific, ethical, and medical questions. These emerging trends and their potential consequences are reviewed here. © 2014 Elsevier B.V.
Yuen B.T.K.,University of California at Davis |
Yuen B.T.K.,California Institute for Regenerative Medicine |
Knoepfler P.S.,University of California at Davis |
Knoepfler P.S.,California Institute for Regenerative Medicine
Cancer Cell | Year: 2013
A host of cancer types exhibit aberrant histone modifications. Recently, distinct and recurrent mutations in a specific histone variant, histone H3.3, have been implicated in a high proportion of malignant pediatric brain cancers. The presence of mutant H3.3 histone disrupts epigenetic posttranslational modifications near genes involved in cancer processes and in brain function. Here, we review possible mechanisms by which mutant H3.3 histones may act to promote tumorigenesis. Furthermore, we discuss how perturbations in normal H3.3 chromatin-related and epigenetic functions may more broadly contribute to the formation of human cancers. © 2013 Elsevier Inc.
Cellular Biomedicine Group Awarded 2.29 Million Grant from the California Institute for Regenerative Medicine CIRM to Fund AlloJoinTM Allogeneic Stem Cell Therapy for Knee Osteoarthritis KOA in the U.S.
News Article | February 27, 2017
SHANGHAI, China and CUPERTINO, Calif., Feb. 27, 2017 (GLOBE NEWSWIRE) -- Cellular Biomedicine Group Inc. (NASDAQ:CBMG) (“CBMG” or the “Company”), a clinical-stage biopharmaceutical firm engaged in the development of effective immunotherapies for cancer and stem cell therapies for degenerative diseases, announced today that the governing Board of the California Institute for Regenerative Medicine (CIRM), California's stem cell agency, has awarded the Company $2.29 million to support pre-clinical studies of AlloJoinTM, CBMG’s “Off-the-Shelf” Allogeneic Human Adipose-derived Mesenchymal Stem Cells for the treatment of Knee Osteoarthritis in the United States. While CBMG recently commenced two Phase I human clinical trials in China using CAR-T to treat relapsed/refractory CD19+ B-cell Acute Lymphoblastic Leukemia (ALL) and Refractory Diffuse Large B-cell Lymphoma (DLBCL) as well as an ongoing Phase I trial in China for AlloJoinTM in Knee Osteoarthritis (KOA), this latest announcement represents CBMG’s initial entrance into the United States for its “off-the-shelf” allogeneic stem cell candidate AlloJoinTM. The $2.29 million was granted under the CIRM 2.0 program, a comprehensive collaborative initiative designed to accelerate the development of stem cell-based treatments for people with unmet medical needs. After the award, CIRM will be a more active partner with its recipients to further increase the likelihood of clinical success and help advance a pre-clinical applicant’s research along a funding pipeline towards clinical trials. CBMG’s KOA pre-clinical program is considered late-stage, and therefore it meets CIRM 2.0’s intent to accelerate support for clinical stage development for identified candidates of stem cell treatments that demonstrate scientific excellence. "We are deeply appreciative to CIRM for their support and validation of the therapeutic potential of our KOA therapy,” said Tony (Bizuo) Liu, Chief Executive Officer of CBMG. “We thank Dr. C. Thomas Vangsness, Jr., in the Department of Orthopaedic Surgery at the Keck School of Medicine of the University of Southern California and Dr. Qing Liu-Michael at the Broad Center for Regenerative Medicine and Stem Cell Research at USC, who helped significantly with the grant application process. The CIRM grant is the first step in bringing our allogeneic human adipose-derived mesenchymal stem cell treatment for knee osteoarthritis (AlloJoinTM) to the U.S. market. Our AlloJoinTM program has previously undergone extensive manufacturing development and pre-clinical studies and is undergoing a Phase I clinical trial in China. In order to demonstrate comparability with cell banks previously produced in China for our U.S. IND filing, we are addressing the pre-clinical answers required for the FDA. With the funds provided by CIRM, we will replicate and validate the manufacturing process and control system at the cGMP facility located at Children’s Hospital Los Angeles to support the filing of an IND with the FDA. The outcome of this grant will enable us to have qualified final cell products ready to use in a Phase I clinical trial with Dr. Vangsness as the Principal Investigator and the Keck School of Medicine of USC as a trial site. Dr. Vangsness is familiar with both stem cell biology and KOA, and has led the only randomized double-blind human clinical study to investigate expanded allogeneic mesenchymal stem cells to date. Our endeavor in the U.S. market will further strengthen our commercialization pipeline.” CBMG recently announced promising interim 3-month safety data from its Phase I clinical trial in China for AlloJoinTM, its off-the-shelf allogeneic stem cell therapy for KOA. The trial is on schedule to be completed by the third quarter of 2017. At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and to act with a sense of urgency commensurate with that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies. With $3 billion in funding and over 280 active stem cell programs in our portfolio, CIRM is the world's largest institution dedicated to helping people by bringing the future of medicine closer to reality. For more information, please visit www.cirm.ca.gov. According to the Foundation for the National Institutes of Health, there are 27 million Americans with Osteoarthritis (OA), and symptomatic Knee Osteoarthritis (KOA) occurs in 13% of persons aged 60 and older. The International Journal of Rheumatic Diseases, 2011 reports that approximately 57 million people in China suffer from KOA. Currently no treatment exists that can effectively preserve knee joint cartilage or slow the progression of KOA. Current common drug-based methods of management, including anti-inflammatory medications (NSAIDs), only relieve symptoms and carry the risk of side effects. Patients with KOA suffer from compromised mobility, leading to sedentary lifestyles; doubling the risk of cardiovascular diseases, diabetes, and obesity; and increasing the risk of all causes of mortality, colon cancer, high blood pressure, osteoporosis, lipid disorders, depression and anxiety. According to the Epidemiology of Rheumatic Disease (Silman AJ, Hochberg MC. Oxford Univ. Press, 1993:257), 53% of patients with KOA will eventually become disabled. Cellular Biomedicine Group, Inc. develops proprietary cell therapies for the treatment of cancer and degenerative diseases. Our immuno-oncology and stem cell projects are the result of research and development by CBMG’s scientists and clinicians from both China and the United States. Our GMP facilities in China, consisting of twelve independent cell production lines, are designed and managed according to both China and U.S. GMP standards. To learn more about CBMG, please visit www.cellbiomedgroup.com. This press release contains forward-looking statements—including descriptions of plans, strategies, trends, specific activities, investments and other non-historical facts—as defined by the Private Securities Litigation Reform Act of 1995, Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking information is inherently uncertain, and actual results could differ materially from those anticipated due to a number of factors, which include risks inherent in doing business, trends affecting the global economy (including the devaluation of the RMB by China in August 2015), and other risks detailed in CBMG’s reports filed with the Securities and Exchange Commission, quarterly reports on form 10-Q, current reports on form 8-K and annual reports on form 10-K. Forward-looking statements may be identified by terms such as "may," "will," "expects," "plans," "intends," "estimates," "potential," "continue" or similar terms or their negations. Although CBMG believes the expectations reflected in the forward-looking statements are reasonable, they cannot guarantee that future results, levels of activity, performance or achievements will be obtained. CBMG does not have any obligation to update these forward-looking statements other than as required by law.
News Article | February 23, 2017
BASKING RIDGE, N.J., Feb. 23, 2017 (GLOBE NEWSWIRE) -- Caladrius Biosciences, Inc. (NASDAQ:CLBS) (“Caladrius” or the “Company”), a cell therapy company combining a select therapeutic development pipeline with an industry-leading development and manufacturing services provider (PCT), announces today that the California Institute for Regenerative Medicine (CIRM) has awarded a grant to Caladrius, providing up to $12.2 million for the development of CLBS03. CLBS03 is the Company’s investigational cell therapy currently being evaluated as a treatment for recent onset type 1 diabetes (T1D) in a Caladrius-sponsored Phase 2 trial, the Sanford Project: T-Rex Study, in collaboration with Sanford Research, a subsidiary of Sanford Health. The grant from CIRM, which was recommended for approval by its distinguished and independent panel of scientific reviewers, is a significant endorsement of the potential for Caladrius’ novel approach for treating T1D with cell therapy by restoring immune balance. The award has important implications as it is expected to fund a significant portion of the remaining cost of the Company’s Phase 2 trial currently underway. The grant will be used to cover expenses including all manufacturing and development based in California and other trial costs dependent upon the proportion of subjects enrolled in California, with consumption of at least $6 million of the award expected. CLBS03 uses the patient’s own regulatory T cells (Tregs) to treat autoimmune disease. Tregs are a natural part of the human immune system that regulate the activity of T effector cells, which are responsible for protecting the body from viruses and other foreign antigens. When Tregs function properly, only harmful foreign materials are attacked by T effector cells. In autoimmune diseases, it is thought that deficient Treg activity permits the T effector cells to attack the body’s own beneficial cells and, in the case of T1D, insulin-producing pancreatic beta cells, thereby reducing and eventually eliminating the body’s ability to produce sufficient amounts of insulin. Caladrius’ novel approach seeks to restore immune balance by augmenting the number and activity of a patient’s own Tregs and using their innate capabilities to modulate multiple facets of the effector arm of the immune system. CLBS03 has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA), and Advanced Therapeutic Medicinal Product classification from the European Medicines Agency. Patients are currently being enrolled in the second cohort of the Phase 2 trial, with an interim analysis of early therapeutic effect expected by the end of 2017. "We are grateful to CIRM and the experts who reviewed and endorsed our application. We firmly believe that this therapy has the potential to improve the lives of people with T1D and this grant helps us advance our Phase 2 clinical study with the goal of determining the potential for CLBS03 to be an effective therapy in this important indication," said David J. Mazzo, PhD, Caladrius’ Chief Executive Officer. "This grant substantiates our approach to identify and secure non-dilutive funding for our development programs and helps position Caladrius as a leader among cell therapy and autoimmune disease therapy developers." About Caladrius Biosciences Caladrius Biosciences, Inc. is a cell therapy development company with cell therapy products in development based on multiple technology platforms and targeting autoimmune and cardiology indications. The company’s subsidiary, PCT, is a leading development and manufacturing partner exclusively focused on the cell therapy industry and has served over 100 clients since 1999. PCT provides a wide range of innovative services including product and process development, GMP manufacturing, engineering and automation, cell and tissue processing, logistics, storage and distribution, as well as expert consulting and regulatory support. For more information on Caladrius please visit www.caladrius.com and for more information on PCT please visit www.pctcaladrius.com. This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements reflect management’s current expectations, as of the date of this press release, and involve certain risks and uncertainties. All statements other than statements of historical fact contained in this press release are forward-looking statements. The Company’s actual results could differ materially from those anticipated in these forward-looking statements as a result of various factors. Factors that could cause future results to materially differ from the recent results or those projected in forward-looking statements include the “Risk Factors” described in the Company’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 15, 2016, and in the Company’s other periodic filings with the SEC. The Company’s further development is highly dependent on, among other things, future medical and research developments and market acceptance, which are outside of its control.
Dimmeler S.,Goethe University Frankfurt |
Ding S.,University of California at San Francisco |
Rando T.A.,Stanford University |
Trounson A.,California Institute for Regenerative Medicine
Nature Medicine | Year: 2014
The scientific community is currently witnessing substantial strides in understanding stem cell biology in humans; however, major disappointments in translating this knowledge into medical therapies are flooding the field as well. Despite these setbacks, investigators are determined to better understand the caveats of regeneration, so that major pathways of repair and regrowth can be exploited in treating aged and diseased tissues. Last year, in an effort to contribute to this burgeoning field, Nature Medicine, in collaboration with the Volkswagen Foundation, organized a meeting with a panel of experts in regenerative medicine to identify the most pressing challenges, as well as the crucial strategies and stem cell concepts that can best help advance the translational regenerative field. Here some experts who participated in the meeting provide an outlook at some of those key issues and concepts. © 2014 Nature America, Inc.
News Article | February 15, 2017
LA JOLLA--(February 14, 2017) Not everyone is Fred Astaire or Michael Jackson, but even those of us who seem to have two left feet have got rhythm--in our brains. From breathing to walking to chewing, our days are filled with repetitive actions that depend on the rhythmic firing of neurons. Yet the neural circuitry underpinning such seemingly ordinary behaviors is not fully understood, even though better insights could lead to new therapies for disorders such as Parkinson's disease, ALS and autism. Recently, neuroscientists at the Salk Institute used stem cells to generate diverse networks of self-contained spinal cord systems in a dish, dubbed circuitoids, to study this rhythmic pattern in neurons. The work, which appears online in the February 14, 2017, issue of eLife, reveals that some of the circuitoids--with no external prompting--exhibited spontaneous, coordinated rhythmic activity of the kind known to drive repetitive movements. "It's still very difficult to contemplate how large groups of neurons with literally billions if not trillions of connections take information and process it," says the work's senior author, Salk Professor Samuel Pfaff, who is also a Howard Hughes Medical Institute investigator and holds the Benjamin H. Lewis Chair. "But we think that developing this kind of simple circuitry in a dish will allow us to extract some of the principles of how real brain circuits operate. With that basic information maybe we can begin to understand how things go awry in disease." Nerve cells in your brain and spinal cord connect to one another much like electronic circuits. And just as electronic circuits consist of many components, the nervous system contains a dizzying array of neurons, often resulting in networks with many hundreds of thousands of cells. To model these complex neural circuits, the Pfaff lab prompted embryonic stem cells from mice to grow into clusters of spinal cord neurons, which they named circuitoids. Each circuitoid typically contained 50,000 cells in clumps just large enough to see with the naked eye, and with different ratios of neuronal subtypes. With molecular tools, the researchers tagged four key subtypes of both excitatory (promoting an electrical signal) and inhibitory (stopping an electrical signal) neurons vital to movement, called V1, V2a, V3 and motor neurons. Observing the cells in the circuitoids in real time using high-tech microscopy, the team discovered that circuitoids composed only of V2a or V3 excitatory neurons or excitatory motor neurons (which control muscles) spontaneously fired rhythmically, but that circuitoids comprising only inhibitory neurons did not. Interestingly, adding inhibitory neurons to V3 excitatory circuitoids sped up the firing rate, while adding them to motor circuitoids caused the neurons to form sub-networks, smaller independent circuits of neural activity within a circuitoid. "These results suggest that varying the ratios of excitatory to inhibitory neurons within networks may be a way that real brains create complex but flexible circuits to govern rhythmic activity," says Pfaff. "Circuitoids can reveal the foundation for complex neural controls that lead to much more elaborate types of behaviors as we move through our world in a seamless kind of way." Because these circuitoids contain neurons that are actively functioning as an interconnected network to produce patterned firing, Pfaff believes that they will more closely model a normal aspect of the brain than other kinds of cell culture systems. Aside from more accurately studying disease processes that affect circuitry, the new technique also suggests a mechanism by which dysfunctional brain activity could be treated by altering the ratios of cell types in circuits. Other authors included: Matthew J. Sternfeld, Christopher A. Hinckley, Niall J. Moore, Matthew T. Pankratz, Kathryn L. Hilde, Shawn P. Driscoll, Marito Hayashi, Neal D. Amin, Dario Bonanomi, Wesley D. Gifford, and Martyn Goulding of Salk; and Kamal Sharma of the University of Illinois, Chicago. The work was funded by the National Cancer Institute at the National Institutes of Health; the Rose Hills Foundation; the H. A. and Mary K. Chapman Charitable Trust; the University of California, San Diego, Neurosciences Graduate Program; a U.S. National Research Service Award fellowship from the U.S. National Institutes of Health National Institute of Neurological Disorders and Stroke; the National Science Foundation; the Japanese Ministry of Education, Culture, Sports, Science, and Technology Long-Term Student Support Program; the Timken-Sturgis Foundation; the California Institute for Regenerative Medicine; the Howard Hughes Medical Institute; the Christopher and Dana Reeve Foundation; the Marshall Heritage Foundation; and the Sol Goldman Charitable Trust. Every cure has a starting point. The Salk Institute embodies Jonas Salk's mission to dare to make dreams into reality. Its internationally renowned and award-winning scientists explore the very foundations of life, seeking new understandings in neuroscience, genetics, immunology, plant biology and more. The Institute is an independent nonprofit organization and architectural landmark: small by choice, intimate by nature and fearless in the face of any challenge. Be it cancer or Alzheimer's, aging or diabetes, Salk is where cures begin. Learn more at: salk.edu.
News Article | March 2, 2017
In June 2016, on the day before his high school graduation, nineteen-year-old Jake Javier suffered a terrible diving accident that left him paralyzed. The incident was covered widely across Bay Area news media and residents quickly mobilized to support Jake and his family. In the March issue of Diablo Magazine, Senior Editor Pete Crooks revisits the story of Jake’s bravery and trailblazing medical care. Crooks caught up with Jake and his parents to discuss life after the accident and explore the life-saving treatments he received at John Muir’s Walnut Creek Medical Center and Santa Clara Valley Medical Center. As part of his therapy, Jake participated in a groundbreaking stem cell procedure by the California Institute for Regenerative Medicine in Oakland. Jake was one of five people in the world to receive an injection of 10 million embryonic stem cells. “This story needed to be revisited.” says Crooks. “Diablo magazine is the town square of the East Bay, and we needed to give Jake’s story time to develop and really share how brave and pioneering he is.” The article details the days and months following the accident and how the community rallied around Jake. Friends, classmates and even strangers joined forces to organize fundraising drives and provide support for the Javier family. To read about the incredible support from the community, Jake’s plans for a future as a college freshman, and view a link to support Jake through the JaviStrong54 Foundation, visit diablomag.com. About Diablo Magazine Covering topics ranging from travel, culture, and personalities to entertainment, recreation, and food, Diablo magazine is written specifically for the San Francisco East Bay market—from Central Contra Costa, into the Oakland and Berkeley hills, and throughout the Tri-Valley. With locally driven editorial content, beautiful photography, and resource listings, Diablo is a unique celebration of the San Francisco East Bay. Published since 1979, Diablo has been recognized for its editorial and design with numerous awards, including previous Maggie Awards for Best Overall Publication and Best Regional and State Magazine in the consumer category. About Diablo Publications For 35 years, Diablo Publications has been creating award-winning publications, including Diablo magazine, Napa Sonoma magazine, Diablo Weddings, the Oakland Visitors' Guide, Diablo Arts, and the Tri-Valley California Visitors Guide. Covering travel, theater, lifestyle, and home design, Diablo Publications celebrates the people, places, and pleasures of the East Bay and North Bay. Diablo Publications’ custom content division, Diablo Custom Publishing (DCP), provides complete print and online marketing communications and customer publishing services for corporate clients nationwide. For more information, visit diablomag.com or dcpubs.com. Diablo Publications is an employee-owned company.
News Article | March 1, 2017
Infants born with a type of the devastating immune disorder SCID, or “bubble boy disease,” may have the option of a novel gene therapy treatment, thanks to a clinical trial at UCSF Benioff Children’s Hospital San Francisco. The trial is funded by a five-year, $11.9-million grant from the California Institute for Regenerative Medicine (CIRM) to test technology developed by St. Jude Children’s Research Hospital that delivers a functional gene into the patient’s blood-producing stem cells. If successful, the gene therapy could provide an alternative to stem cell transplants using donor cells, which can result in serious infection. The trial expects to treat up to 15 children over the next five years and is open to patients with X-linked severe combined immunodeficiency disease (X-linked SCID), which affects only males. This is the most common form of SCID, which occurs in 1 in every 60,000 newborns, and is caused by defects in the functioning of lymphocytes – the white blood cells that are the advanced fighting forces of the immune system. Babies born with SCID appear normal at birth but become sick from infections, skin rashes and failure to gain weight at 3-to-6 months of age. Without a stem cell transplant, they may die before their first birthday. “What is unique about this trial is that the patient’s own bone marrow stem cells are collected and corrected with the gene therapy, and the corrected cells are then reinfused into the patient,” said Morton Cowan, M.D., of the UCSF Division of Allergy, Immunology, and Blood and Marrow Transplant, and principal investigator of the trial at UCSF. “In stem cell transplants from a donor other than the patient, up to 20 percent of patients with SCID will develop graft-versus-host disease, in which the donor cells attack the recipient’s tissues. In addition, there is always a risk of the recipient rejecting the donor cells,” Cowan said. “Using the patient’s own stem cells means no rejection and no graft-versus-host disease.” The bone marrow transplant program at UCSF is among the largest SCID transplant centers in North America. UCSF pediatric immunologist Jennifer Puck, M.D., is known for pioneering the SCID screening method and for nominating SCID to a federal advisory committee for inclusion in the newborn screening panel. Since the screen became available in California in 2010, UCSF has treated more than 30 infants diagnosed with SCID by newborn screening. UCSF also played an instrumental role in the St. Jude treatment protocol by including a targeted chemotherapy agent, busulfan, along with the gene therapy, which is expected to optimize immune correction. While previous trials have tested gene therapy for this condition, they did not combine it with chemotherapy and had only partial immune correction. Since a low dose of the medication is used, short- and long-term effects are expected to be minimized. Three patients already have been treated with this lentiviral gene therapy vector – two at St. Jude and one at UCSF. The transduction process, in which genetic material is transferred via vector, currently takes place at St. Jude, which freezes the transduced cells and returns them to UCSF for infusion into the patient. The CIRM funding will enable UCSF to begin doing transductions using the St. Jude vector at the UCSF Pediatric Cell Therapy Laboratory, as well as covering the cost of treating patients in the trial. “We believe this trial will not only help us understand more about how lentiviral gene therapy works, but how the use of low-dose busulfan potentially will be effective in treating other non-malignant diseases like sickle-cell anemia, chronic granulomatous disease, marrow failure syndromes and even some cancers in which the patient is too ill to undergo the more toxic traditional treatments,” said Cowan. “It will also give us a better idea of what toxicities may be associated with the use of these new vectors, in particular whether they are indeed safer than the older, gamma-retroviral vectors that were associated with a high risk of leukemia, seen in early gene therapy trials for X-linked SCID and other primary immune deficiencies.”
Trounson A.,California Institute for Regenerative Medicine |
Shepard K.A.,California Institute for Regenerative Medicine |
DeWitt N.D.,California Institute for Regenerative Medicine
Current Opinion in Genetics and Development | Year: 2012
In the past few years, cellular programming, whereby virtually all human cell types, including those deep within the brain or internal organs, can potentially be produced and propagated indefinitely in culture, has opened the door to a new type of disease modeling. Importantly, many diseases or disease predispositions have genetic components that vary from person to person. Now cells from individuals can be readily reprogrammed to form pluripotent cells, and then directed to differentiate into the lineage and the cell type in which the disease manifests. Those cells will contain the genetic contribution of the donor, providing an excellent model to delve into human disease at the level of individuals and their genomic variants. To date, over fifty such disease models have been reported, and while the field is young and hurdles remain, these tools promise to inform scientists about the cause and cellular-molecular mechanisms involved in pathology, unravel the role of environmental versus hereditary factors driving disease, and provide an unprecedented tool for screening therapeutic agents that might slow or halt disease progression. © 2012 Elsevier Ltd.
Trounson A.,California Institute for Regenerative Medicine |
Grieshammer U.,California Institute for Regenerative Medicine
Cell | Year: 2012
In this issue, Tachibana et al. report the generation of the first chimeras from a nonhuman primate, the rhesus monkey. Unlike mice, rhesus chimeras fail to form when embryonic stem cells are injected into blastocysts. Instead, chimera formation is achieved by aggregation of several four-cell embryos. © 2012 Elsevier Inc.