Neurological Disorders

Coding Dimension ID: 
303
Coding Dimension path name: 
Neurological Disorders
Grant Type: 
Late Stage Preclinical Projects
Grant Number: 
CLIN1-11404
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$5 666 077
Disease Focus: 
Neurological Disorders
Spina Bifida
Human Stem Cell Use: 
Adult Stem Cell
Public Abstract: 

Therapeutic Candidate or Device

Allogeneic Placenta-derived Mesenchymal Stem Cells Seeded on Cook Biodesign® Dural Graft Extracellular Matrix (PMSC-ECM)

Indication

Myelomeningocele (MMC) -or Spina Bifida -diagnosed prenataly

Therapeutic Mechanism

Placenta-derived mesenchymal stem cells (PMSCs) act by a paracrine mechanism, secreting a variety of growth factors, cytokines, and extracellular vesicles. This secretory profile is unique to PMSCs and is responsible for protecting motor neurons from apoptosis, which occurs due to chemical and mechanical trauma when motor neurons are exposed to the intrauterine environment. PMSC treatment increases the density of motor neurons in the spinal cord, leading to improved motor function.

Unmet Medical Need

The current standard of care in utero surgery, while promising, still leaves 58% of patients unable to walk independently. There is an extraordinary need for a therapy that prevents or lessens the severity of the devastating and costly lifelong disabilities associated with the disease.

Project Objective

IND filing, Phase 1/2 trial start-up activities

Major Proposed Activities

  • Manufacture product to supply the proposed studies and clinical trial
  • Assess safety of the therapeutic PMSC product
  • Assess efficacy using clinical-grade product
Statement of Benefit to California: 

There is a high incidence of MMC in CA with 39.1% of the population being of Hispanic or Latino descent, a demographic that is affected by MMC at a disproportionately high rate. The cost to CA is approximately $532,000 per child, but for many, the cost may be several million dollars due to ongoing treatment. Indirect costs include pain and suffering, specialized childcare, and lost time of unpaid caregivers. A therapy for MMC would relieve the tremendous emotional and economic cost burden to CA.

Grant Type: 
Late Stage Preclinical Projects
Grant Number: 
CLIN1-11059
Investigator: 
ICOC Funds Committed: 
$5 811 340
Disease Focus: 
Neurological Disorders
Parkinson's Disease
Human Stem Cell Use: 
Adult Stem Cell
Public Abstract: 

Therapeutic Candidate or Device

CNS10-NPC-GDNF is a neural progenitor cell line transfected with glial cell line derived neurotrophic factor (GDNF)

Indication

Mid-stage Parkinson's disease (UPDRS stage III or lower)

Therapeutic Mechanism

Degeneration of dopaminergic neurons that project from the substantia nigra to the striatum causes the primary motor symptoms of Parkinson's disease. CNS10-NPC-GDNF cells will be transplanted into the putamen, and are expected undergo limited migration to areas of degeneration, induce sprouting of dopaminergic terminals and protect dopamine cell bodies. The cells can mature into astrocytes that may provide additional protection of degenerating regions through secretion of supportive factors.

Unmet Medical Need

Current treatments provide symptomatic relief of Parkinson's disease (PD), but become less effective over time as they have no effect on the disease process. CNS10-NPC-GDNF is expected to slow the disease progression by inducing sprouting of dopaminergic terminals and protecting dopaminergic cells.

Project Objective

Complete pre-clinical studies, and file an IND.

Major Proposed Activities

  • Manufacture of CNS10-NPC-GDNF to supply the proposed clinical trial
  • Demonstrate longterm lack of tumorigenicity and safety in rats
  • Demonstrate safety and tolerability of CNS10-NPC-GDNF in aged MPTP lesioned non-human primate model of Parkinson's disease
Statement of Benefit to California: 

Parkinson's disease is a debilitating disease, which puts a huge burden on state resources through the need for care givers and medical care. While primarily an effort to reduce patient and family suffering, this project will also ease the cost of caring for PD patients in California if successful. This in turn will attract scientists, clinicians, and biotech companies to the state of California thus increasing state revenue and state prestige in this rapidly growing field.

Grant Type: 
Late Stage Preclinical Projects
Grant Number: 
CLIN1-10953
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$6 000 000
Disease Focus: 
Huntington's Disease
Neurological Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 

Therapeutic Candidate or Device

The therapeutic candidate is a human Neural Stem Cell product to prevent or delay disease symptoms for treatment of Huntington's disease (HD).

Indication

Huntington’s disease, a progressive, degenerative brain disease, typically strikes in midlife with no disease modifying treatment
treatments exist.

Therapeutic Mechanism

Based on our pre-clinical studies, the human neural stem cells engraft and differentiate into neuronal populations, express the neurotrophic factor BDNF and reduce mutant Huntingtin protein accumulation. Further, host tissue forms synaptic contacts with transplanted cells and may provide new and functional connections to reduce the aberrant cortical excitability in HD. These molecular and histological improvements correlate with improvement in behavior and electrophysiological deficits.

Unmet Medical Need

No treatment currently exists that can slow or prevent the unrelenting progression of Huntington’s disease, a devastating brain disease,
therefore a completely unmet medical need exists.

Project Objective

File an Investigational New Drug request with FDA.

Major Proposed Activities

  • Good Manufacturing Practice (GMP) manufacturing and characterization of the cell product to supply the first in human study.
  • Good laboratory practice (GLP) long term safety, biodistribution and tumorigenicity studies in HD modeled and Wt mice.
  • Investigational New Drug (IND) preparation, publishing and submission .
Statement of Benefit to California: 

The disability, loss of personal freedom and earning potential, and costly institutional care of HD is devastating. Developing a therapeutic product will allow patients to live independently longer after diagnosis, and result in saving considerable costs for healthcare and caregiving, and extending the quality of life for HD patients and their family members. It will also benefit California through new technologies and intellectual property resulting in possible job creation and revenues.

Grant Type: 
Late Stage Preclinical Projects
Grant Number: 
CLIN1-09433
Investigator: 
Institution: 
Type: 
PI
ICOC Funds Committed: 
$5 300 000
Disease Focus: 
Neurological Disorders
Stroke
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 

Therapeutic Candidate or Device

A human embryonic derived, non-genetically modified neural stem cell (NR1), originally derived from the Wi-Cell H-9 line.

Indication

Patients with chronic motor deficits, from 6 months to 5 years after stroke. NR1 cells will be transplanted into the brain near the stroke.

Therapeutic Mechanism

The proposed therapeutic mechanism of action of NR1 neural stem cells is the secretion of factors that enhance the brain’s own ability to heal itself after stroke, including the creation of new blood vessels to replace those that were injured beyond repair, and modulation the immune system.

Unmet Medical Need

Stroke is the leading cause of adult disability. There is no medical therapy that promotes stroke recovery, establishing this as a major unmet medical need. NR1 will be the first stem cell-derived therapy directed towards improving disability in this disease.

Project Objective

To prepare and submit an IND application.

Major Proposed Activities

  • Completion of ongoing in vitro work.
  • Animal selection for and carrying out GLP toxicology and biodistribution studies of the transplanted NR1 cells into the rodent brain.
  • Pre-IND activities leading to submission of the IND.
Statement of Benefit to California: 

This program provides several areas of benefit to California as the first stem cell-derived therapy for recovery of function after stroke. It will provide medical benefit by directly treating disabled Californians. It will provide economic benefit in a medical therapy that is manufactured and tested within the state. It will provide scientific benefit by pioneering the science of brain repair in California universities, with likely spin-off of additional novel therapies for neurological disease.

Grant Type: 
Preclinical Development Awards
Grant Number: 
PC1-08117
Investigator: 
Type: 
PI
ICOC Funds Committed: 
$4 951 623
Disease Focus: 
Huntington's Disease
Neurological Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
Public Abstract: 

Huntington’s disease (HD) is a devastating degenerative brain disease with at least a 1 in 10,000 prevalence that inevitably leads to death. These numbers do not fully reflect the large societal and familial cost of HD, which requires extensive care-giving. HD has no effective treatment or cure and symptoms unstoppably progress for 15-20 years, with onset typically striking in midlife. Because HD is genetically dominant, the disease has a 50% chance of being inherited by the children of patients. Symptoms of the disease include uncontrolled movements, difficulties in carrying out daily tasks or continuing employment, and severe psychiatric manifestations including depression. Current treatments only address some symptoms and do not change the course of the disease, therefore a completely unmet medical need exists. Human embryonic stem cells (hESCs) and their derivatives offer a possible long-term treatment approach that could relieve the tremendous suffering experienced by patients and their families. HD is the 3rd most prevalent neurodegenerative disease, but because it is entirely genetic and the mutation known, a diagnosis can be made with certainty and clinical applications of hESCs may provide insights into treating brain diseases that are not caused by a single, known mutation. Trials in mice where protective factors were directly delivered to the brains of HD mice have been effective, suggesting that delivery of these factors by hESCs may help patients. Transplantation of tissue in HD patients suggests that replacing neurons that are lost may also be effective. The ability to differentiate hESCs into neural populations offers a powerful and sustainable alternative to provide neuroprotection to the brain with the possibility of cell replacement. We have assembled a multidisciplinary team of investigators and consultants with expertise in basic, translational and clinical development and have identified a lead developmental candidate, ESI-017 neural stem cells, that have disease modifying activity in HD mice with sufficient promise to perform systematic efficacy and safety studies in HD mice with cells generated for this project. We will utilize the collaborative research team, additional preclinical and clinical investigators, stem cell experts and FDA consultants to finalize work that will lead to a productive pre-IND meeting with the FDA and a path forward for clinical trials with the neural stem cell development candidate.

Statement of Benefit to California: 

The disability and loss of earning power and personal freedom resulting from Huntington's disease (HD) is devastating and creates a financial burden for California. Individuals are struck in the prime of life, at a point when they are their most productive and have their highest earning potential. As the disease progresses, individuals require institutional care at great financial cost. Therapies using human embryonic stem cells (hESCs) have the potential to change the lives of hundreds of individuals and their families, which brings the human cost into the thousands. For the potential of hESCs in HD to be realized, we have brought together a team of investigators highly experienced in HD basic science and preclinical development, stem cell research, HD clinical trials and FDA regulatory activities to evaluate a human stem cell derived neural stem cell line, ESI-107 NSC in HD mouse models. This selection of this development candidate is based on efficacy in behavioral and electrophysiology measurements in a rapidly progressing mouse model of HD. HD is the 3rd most prevalent neurodegenerative disease, but because it is entirely genetic and the mutation known, a diagnosis can be made with certainty and clinical applications of NSCs may provide insights into treating brain diseases that are not caused by a single, known mutation. We have assembled a strong team of California-based investigators to carry out proposed studies to move ESI-017 NSCs to the point of a productive pre-IND meeting with the FDA to ultimately move this clinical product into Investigative New Drug-enabling (IND) activities with the goal of performing clinical trials in HD subjects. Anticipated benefits to the citizens of California include: 1) development of new human stem cell-based treatments for HD with application to other neurodegenerative diseases such as Alzheimer's and Parkinson's diseases that affect thousands of individuals in California; 2) improved methods for following the course of the disease in order to treat HD as early as possible before symptoms are manifest; 3) transfer of new technologies and intellectual property to the public realm with resulting IP revenues coming into the state with possible creation of new biotechnology spin-off companies; and 4) reductions in extensive care-giving and medical costs. It is anticipated that the return to the State in terms of revenue, health benefits for its Citizens and job creation will be substantial.

Grant Type: 
Preclinical Development Awards
Grant Number: 
PC1-08103
Investigator: 
Type: 
PI
Name: 
Type: 
Co-PI
ICOC Funds Committed: 
$2 184 032
Disease Focus: 
Neurological Disorders
Pediatrics
Spina Bifida
Human Stem Cell Use: 
Adult Stem Cell
Cell Line Generation: 
Adult Stem Cell
Public Abstract: 

Myelomeningocele – also known as spina bifida – is a devastating and costly defect that causes lifelong paralysis as well as bowel and bladder incontinence in newborns. It is one of the most common birth defects worldwide, with four children in the United States born with spina bifida every day. Spina bifida affects the physical, educational, social, and psychological development of these children. Most patients require multiple surgeries and hospitalizations throughout their lives. Physicians are now able to diagnose this disease during pregnancy, and new fetal surgical techniques allow surgeons to safely operate on these children in the womb. This unique fetal surgery was studied in the award winning Management of Myelomeningocele Study (MOMS). The MOMS trial showed – for the first time ever – that the paralysis associated with spina bifida could be improved. Children treated in the womb were more likely to walk independently than those who were repaired after birth. However, the improvements seen were not perfect and the majority of children treated with fetal surgery still had some level of paralysis or lower extremity weakness.

Our research has built upon the success of the MOMS trial to address the residual deficits seen in children even after treatment with fetal surgery. We have developed a placental stem cell based therapy that can be applied at the time of fetal repair, in order to reverse spinal cord damage. After six years of laboratory research investigating different stem cell types and the best way to deliver a stem cell based treatment in the womb, we have discovered a placental stem cell therapy that cures spina bifida in the animal model. Animals treated with these cells can make a full recovery and are able to walk normally without any evidence of lower extremity paralysis. These amazing results require additional testing and FDA approval before the therapy can be used in humans. With this proposal, we will optimize this stem cell product, validate its effectiveness, determine the optimal dose, and confirm its preliminary safety in order to translate this new treatment to clinical trials. Stem cell therapy for spina bifida could cure this devastating disease, alleviating a massive burden on children, families, and society.

Statement of Benefit to California: 

Spina bifida is one of the most common, costly, and disabling birth defects. Within the United States, four children per day are born with this devastating disease. In California, the 5-year statewide incidence of spina bifida was 6.8 cases per 10,000 live births between 1999 and 2003, significantly higher than the Healthy People 2010 target of 3 per 10,000 births. Additionally, spina bifida disproportionately affects Americans of Hispanic and Latino descent, who make up 37.6% of California’s population. Given the disproportionately high incidence of children born with spina bifida in California, and the lifelong disability these children live with, spina bifida is a substantial economic burden to the state. The estimated average total lifetime cost to California is approximately $532,000 for each child born with spina bifida. However, for many children, the cost may be several million dollars due to repeat surgical procedures, frequent hospitalizations, and the need for ongoing physical and cognitive rehabilitation. In addition to the direct medical costs associated with spina bifida, the indirect costs include: pain and suffering, cost of specialized childcare, and the lost earning potential of unpaid caregivers, which compound the impact the disease has on California’s economy.

There is currently no cure for spina bifida, and interventions that mitigate the negative consequences of the disease (lower body paralysis, bowel and bladder incontinence) are urgently needed. For the first time, hope for an improved treatment option was provided by the award winning Management of Myelomeningocele Study (MOMS). The MOMS trial was a multicenter randomized controlled trial demonstrating that the paralysis associated with spina bifida might be improved by surgical repair of the defect before birth. This promise of fetal intervention for spina bifida was based on the hypothesis that early in utero treatment would have the potential to fix the defect before permanent spinal cord damage occurred. While the MOMS trial did demonstrate an improvement in the lower extremity paralysis of those patients undergoing in utero repair compared to postnatal repair, these improvements were not universal for all children. This proposal presents an innovative placental stem cell-based therapy to augment fetal repair and further improve and possibly cure the devastating and costly neurologic deficits of spina bifida. A cure for spina bifida would relieve California families and society of the tremendous emotional and economic cost burden of this debilitating disease, and would be life changing for future children afflicted with spina bifida.

Grant Type: 
Preclinical Development Awards
Grant Number: 
PC1-08086
Investigator: 
ICOC Funds Committed: 
$1 737 271
Disease Focus: 
Alzheimer's Disease
Neurological Disorders
Human Stem Cell Use: 
Embryonic Stem Cell
iPS Cell
Cell Line Generation: 
Embryonic Stem Cell
iPS Cell
Public Abstract: 

Over 6 million people in the US suffer from Alzheimer’s disease (AD). There are no drugs that prevent the death of nerve cells in AD, nor has any drug been identified that can stimulate nerve cell replacement in aged human brain. Importantly, even if nerve cells could be replaced, the toxic environment of the AD brain which caused the disease in the first place will likely kill any cells that are born into that environment unless they are resistant to those conditions or can be protected by a drug. Therefore, drugs that stimulate the generation of new neurons (neurogenesis) alone will not be effective. A drug with both neurogenic and neuroprotective properties is required. With the ability to use cells derived from human neural precursor cells (hNPCs) derived from human embryonic stem cells (hESCs) as a screen for neurogenic compounds, we have shown that it is possible to identify and tailor drugs for therapeutic use in AD. With the support of CIRM, we have recently made a very potent AD drug candidate that is exceptionally effective in promoting the making of new nerve cells from human embryonic stem cells. It is both neurogenic and has therapeutic efficacy in a rodent model of AD. However, this molecule needs more preclinical development work before it can start the formal FDA pre clinical toxicity screening protocols. This work will optimize the chances for its true therapeutic potential in AD, and presents a unique opportunity to expand the use of hESCs for the development of a therapeutic for a disease for which there is no cure.

Statement of Benefit to California: 

Over 6 million people in the US suffer from AD, and unless a viable therapeutic is identified it is estimated that this number will increase to at least 16 million by 2050, with a cost of well over $1 trillion per year, likely overwhelming both the California and national health care systems. There is no treatment to prevent, cure or slow down this condition. In this application we have used the new human stem cell technologies to develop an AD drug candidate that stimulates the multiplication of nerve precursor cells derived from human embryonic stem cells. This approach presents a unique opportunity to expand the use of human embryonic stems cells for the development of a therapeutic for a disease for which there is no cure, and could lead to a paradigm shift in the treatment of neurodegenerative disease. Since our AD drug discovery approach is fundamentally different from the unsuccessful approaches used by the pharmaceutical industry. It could also stimulate new biotech. The work in this proposal addresses one of the most important medical problems of California as well as the rest of the world, and if successful would benefit all.

Grant Type: 
Tools and Technologies III
Grant Number: 
RT3-07948
Investigator: 
Institution: 
Type: 
PI
Institution: 
Type: 
Co-PI
ICOC Funds Committed: 
$1 452 708
Disease Focus: 
Neurological Disorders
Spinal Cord Injury
Human Stem Cell Use: 
iPS Cell
Public Abstract: 

One critical bottleneck in the translation of regenerative medicine into the clinic is the efficient delivery and engraftment of transplanted cells. While direct injection is the least invasive method for cell delivery, it commonly results in the survival of only 5-20% of cells. Studies suggest that delivery within a carrier gel may enhance cell viability, but most of the gels used previously were naturally derived materials that have complex and unknown compositions. In our previous CIRM-funded work, we discovered that pre-encapsulating cells in very weak hydrogels offers the best protection during injection; however, those gels may be too compliant to support long-term cell survival. To address these limitations, we propose the design of a fully defined, customizable, and injectable material that initially forms a weak gel that then stiffens post-injection. We focus our studies on the delivery of human induced pluripotent stem cell-derived neural progenitors for the treatment of spinal cord injury (SCI). There are ~12,000 new SCI patients in the US each year, primarily young adults. SCI commonly results in paralysis, and the estimated lifetime cost for a patient can rise above $4 million dollars. In preclinical models of SCI, stem cell therapies have resulted in partial regeneration; however, reproducible delivery and engraftment of sufficient cells remain difficult and unmet challenges. This award potentially develops transformational regenerative therapies for SCI.

Statement of Benefit to California: 

The annual incidence of spinal cord injuries (SCI) in the United States is estimated at 12,000 new cases per year, with motor vehicle crashes accounting for up to a third of these cases. SCI has devastating impacts not only on the quality of life for the victims and their families, but also on their economic security – the estimated lifetime cost of an SCI patient can rise to over $4 million dollars depending on the severity and age at which the injury was sustained, not including the loss of wages and productivity. Although the most prevalent types of SCIs are those sustained at either the cervical or thoracic vertebrae, there are currently no definitive therapies approved for the chronic management of these SCI. Stem cell-based therapies have recently been shown to be mildly successful in several clinical and pre-clinical trials in various injuries and diseases, and a number of trials are ongoing for applications in SCI. In our proposal, we seek to advance the stem cell-based approach to the treatments of SCI. The potential benefit of this proposal to the state of California and its citizens include 1) the provision of a better medical prognosis for patients with spinal cord injuries, 2) the improved quality of life for SCI patients and their families, 3) the reduction of the burden of health care costs, 4) the creation and maintenance of jobs in the stem cell technology field, and 5) preserving California’s prominence in the field of stem cell research.

Grant Type: 
Tools and Technologies III
Grant Number: 
RT3-07914-B
Investigator: 
Name: 
Institution: 
Type: 
PI
ICOC Funds Committed: 
$1 818 751
Disease Focus: 
Intestinal Disease
Metabolic Disorders
Neurological Disorders
Pediatrics
Human Stem Cell Use: 
Adult Stem Cell
Public Abstract: 

The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have a poor quality of life, and the cost of medical care is over $200,000 per year for each patient. Stem cell therapies offer potential cures for these patients while avoiding the risks of invasive procedures and hazardous treatments. A novel approach to treat these patients is to use stem cells derived from the patient’s own skin to generate the neural system. This has been shown to be feasible in small animals, and the next step hinges on the demonstration of these results in a large animal model of intestinal dysfunction. We will develop a model in large animals that can be used to test the ability of skin-derived stem cells to form the neural system. Skin-derived stem cells will be isolated from large animal models and human skin to demonstrate their potential to generate a functional neural system. These cells will be transplanted into the animal model to determine the best way for these cells to make the intestine function properly. This research will gather critical information needed to begin a clinical trial using skin-derived cells to treat intestinal dysfunction.

Statement of Benefit to California: 

Gastrointestinal dysfunction destroys the lives of thousands of Californians. These Californians have frequent and prolonged hospitalizations and lost wages due to their chronic illness. It is estimated that the health care cost of Californians with gastrointestinal neuromuscular dysfunction is over 400 million dollars annually. Currently, most of these patients are covered by the state’s insurance agency. Stem cell therapies offer potential cures for these patients and reduce this economic burden. The proposed research will obtain critical information needed to begin a clinical trial using skin-derived cells to treat patients with intestinal dysfunction. The California economy will significantly benefit from this research through the reduced costs for health care and increased quality of life of the affected Californians. Additionally, this work will add to the state’s growing stem cell industry and will increase employment opportunities and revenue by the state of California. The educational benefit to Californians involved in this research project will also maintain California’s position in leading the stem cell effort in the future.

Grant Type: 
Tools and Technologies III
Grant Number: 
RT3-07914-A
Investigator: 
Name: 
Type: 
PI
ICOC Funds Committed: 
$1 818 751
Disease Focus: 
Intestinal Disease
Metabolic Disorders
Neurological Disorders
Pediatrics
Human Stem Cell Use: 
Adult Stem Cell
Public Abstract: 

The intestine performs the essential function of absorbing food and water into the body. Without a functional intestine, children and adults cannot eat normal meals, and these patients depend on intravenous nutrition to sustain life. Many of these patients do not have a neural system that coordinates the function of the intestine. These patients have a poor quality of life, and the cost of medical care is over $200,000 per year for each patient. Stem cell therapies offer potential cures for these patients while avoiding the risks of invasive procedures and hazardous treatments. A novel approach to treat these patients is to use stem cells derived from the patient’s own skin to generate the neural system. This has been shown to be feasible in small animals, and the next step hinges on the demonstration of these results in a large animal model of intestinal dysfunction. We will develop a model in large animals that can be used to test the ability of skin-derived stem cells to form the neural system. Skin-derived stem cells will be isolated from large animal models and human skin to demonstrate their potential to generate a functional neural system. These cells will be transplanted into the animal model to determine the best way for these cells to make the intestine function properly. This research will gather critical information needed to begin a clinical trial using skin-derived cells to treat intestinal dysfunction.

Statement of Benefit to California: 

Gastrointestinal dysfunction destroys the lives of thousands of Californians. These Californians have frequent and prolonged hospitalizations and lost wages due to their chronic illness. It is estimated that the health care cost of Californians with gastrointestinal neuromuscular dysfunction is over 400 million dollars annually. Currently, most of these patients are covered by the state’s insurance agency. Stem cell therapies offer potential cures for these patients and reduce this economic burden. The proposed research will obtain critical information needed to begin a clinical trial using skin-derived cells to treat patients with intestinal dysfunction. The California economy will significantly benefit from this research through the reduced costs for health care and increased quality of life of the affected Californians. Additionally, this work will add to the state’s growing stem cell industry and will increase employment opportunities and revenue by the state of California. The educational benefit to Californians involved in this research project will also maintain California’s position in leading the stem cell effort in the future.

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