News - Research
Tay-Sachs Gene Therapy Consortium - Progress Update Highlights- July 2010
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- Twelve GM2 cats have now been treated with gene therapy
- An untreated GM2 cat normally dies at 4.5 months - the oldest treated cat is now over six months old and is highly functional
- The second oldest cat is 5 months old and doing very well - the remaining 10 cats are between 1 and 3 months of age.
- The two older cats do have some hind leg weakness and minor gait abnormalities - but the treatment has made a huge positive difference
- This is very good news for the large animal model and hopes of human clinical trials
- In June two Tay-Sachs sheep were treated with gene therapy
- The sheep tolerated the injections well and are being evaluated
- The Tay-Sachs natural history study is completed and is in the final stages for submission
Tay-Sachs Gene Therapy Consortium
Progress Update - July 2010
1. We have now injected 12 GM2 kittens with AAV vectors encoding the feline subunitsof hexosaminidase. Also, due to recent substantial increases in colony productivity, we have begun treating cats for year 2 of the NIH-funded project ahead of schedule.
The oldest treated GM2 cat is now > 6 months old, and the second oldest is almost 5 months old. The other 10 kittens range between 1 and 3 months of age. Untreated GM2 kittens survive until ~ 4.5 months of age and by the end have a severe whole body tremor and are unable to support their weight on four limbs. In contrast, the oldest treated GM2 cat is able to move around easily, including running after toys and making quick stops and changes in direction. It is also under these conditions that her major clinical symptom becomes apparent: instability/weakness in the rear legs. A new piece of sophisticated equipment to analyze gait in great detail has shown some mild abnormalities in the treated cat, especially on the left side. Nevertheless, there are no apparent balance difficulties when sitting or standing, contrary to what is observed in untreated GM2 kittens at later stages of disease. It is important to note that, although clinical disease is evident in the rear legs, this cat is 1.5 months older than the expected life span for an untreated GM2 cat and is doing very well. In addition, no progression of clinical signs has been noted in this 6 month-old AAV-treated cat
for the past 5 weeks.
The 5-month old treated GM2 cat has very slight rear leg weakness, no balance
difficulties and no body tremor. Once again, we have noted no clear progression of
clinical signs for the past 4-6 weeks.
The younger 1-3 month-old AAV-treated GM2 kittens appear normal thus far, but it is too early to tell whether we will see the same or better results than in the two older cats. Treated and untreated GM2 cats are being subjected to numerous tests including neurological exams, MRI, gait analysis and biochemical analysis of cerebrospinal fluid to determine the extent of disease progression/correction.
2. Four Tay-Sachs sheep along with mothers and siblings arrived at one of the TSGT's member institutions at 5 a.m. on April 17, 2010. At weaning, the mothers and siblings were transported back to their home in Texas. During an extremely busy spring season, we completed the following steps necessary to begin pilot studies of AAV gene therapy in affected sheep, the first clinically relevant model of Tay-Sachs Disease: (1) DNA testing of >50 sheep to identify those affected with Tay-Sachs Disease, carriers and normals; (2) Generation of AAV vectors carrying the sheep alpha and beta genes (the beta gene having to be cloned for the first time) to minimize immune responses in treated sheep; (3) Testing of the AAV vectors in cultured cells to confirm functionality; (4) Initiation of controlled studies of disease progression in untreated sheep, which will allow us to assess whether treatment has had any impact on disease course; (5) Careful definition of the brain injection coordinates necessary to treat Tay-Sachs sheep with AAV gene therapy. Challenges were encountered in this process because of the wide variability in Jacob sheep, which may have from 2 to 6 horns, making standardization of injection coordinates impossible. We learned that each sheep must undergo an MRI prior to surgery for accurate identification of injection coordinates. In addition, a boardcertified veterinary anesthesiologist and an external collaborator from the University of Tennessee were brought in to ensure successful injection of these extremely valuable sheep.
To date, we have learned that disease onset in affected sheep begins at 1-2.5 months of age with occasional stumbling due to "knuckling" of the front hooves. There is mild variability of disease onset and progression. For example, the youngest TS sheep also has the most significant clinical disease, with obvious front limb gait defects and a tendency to lie down much more frequently than the other affected or normal sheep. By contrast, a second TS sheep born 1 week prior to the most severely affected sheep has very mild clinical disease (an almost imperceptible front limb gait defect - "walking down in the fetlock"). The remaining 2 TS sheep began to show clinical signs at similar ages (~8 weeks) and have been treated with AAV gene therapy (see further description below). To date, TS sheep have shown no abnormalities in general health measures (weight, temperature, heart rate), routine blood work (complete blood count, serum chemistries), MRI, ophthalmology exams or response to anesthesia. Other than the gait defects, the only obvious difference in TS sheep is their outgoing / curious nature compared to normal siblings.
In early June, two of the four Tay-Sachs sheep were treated by injection of large amounts of gene therapy vectors into the brain. One sheep was treated with vectors expressing both the Hex alpha and beta subunits while the other sheep was treated with a vector expressing the alpha subunit alone. This experiment should provide valuable information regarding the need for co-expression of both subunits in human clinical trials or whether treatment with a single subunit will be sufficient. Both sheep tolerated the injection procedure well and are at similar mild stages of disease progression currently. Initial tests to evaluate vector function and therapeutic effect are underway.
3. Theretrospective natural history study has been concluded and a manuscript is in the final stages of preparation for submission. A clinical rating scale (CRS) has been developed based on this study. In addition MRI/MRS studies in LOTS patients are underway, and have already generated some rather interesting findings that could have implications for using MRS to measure certain metabolites in the brain of TS patients. An MRI rating scale is also being developed over the summer. Our next goal is to conduct prospective studies to validate imaging and clinical scoring scales and possibly develop new bio-markers that will be used to assess treatment effects in the clinical trial.
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Research Developments - June 2010
In April, the NTSAD Research Initiative Committee announced 5 new grants totaling one-half million dollars for 2010. This is the largest dollar amount that has been funded in the Research Initiative's history and is the first time that multi-year grants have been awarded: 4 of the 5 grants are two-year grants. The grants are as follows:
Jean-Pyo Lee, PhD / Evan Y. Snyder, MD, PhD (Burnham Medical Research Institute): The Therapeutic Potential of Human Induced Pluripotent Stem Cells (IPSCs) in the Sandhoff Disease Mouse Model of Lysosomal Storage Disorders.
- Description: This cutting-edge project will investigate the mechanisms of a new type of stem cells: induced pluripotent stem (IPS) cells.
- Benefit: If IPS derived cells could be used for therapy that would be a major step forward, as it takes away the problem of tissue rejection and the problem of working with embryonic stem cells.
Maria Traka, PhD (University of Chicago): Development of an in vitro approach to identify molecular pathways of Canavan disease
- Description: The investigator will use a mouse model of Canavan disease to study the molecular pathways in Canavan disease.
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Benefit: The project has a high likelihood of enhancing our knowledge of Canavan disease which will help develop therapies in the future.
Fran Platt, PhD (University of Oxford): Optimizing the Therapeutic Potential of Anti-inflammatory Therapy in Tay-Sachs and Related Diseases: Targeting IL-1ß Generated by Aberrant NLRP3 Inflammasome Activation
- Description: Develop a mouse model of Sandhoff disease that lacks a particular receptor and use various analyses to determine the degree of anti-inflammatory benefit achieved in these mice, which will define the mechanism that triggers inflammation.
- Benefit: This grant addresses a significant problem involving ganglioside-associated neuro-inflammation. Clinical trials could proceed quickly if confirmation of a particular inflammatory mechanism can be confirmed.
Gustavo Maegawa, PhD (Johns Hopkins University) Developing a High Throughput Screening Assay to Identify Potential Drugs for Metachromatic Leukodystrophy
- Description: This grant is based on the principle that small molecules can enhance residual enzyme activity in patients with lysosomal storage diseases (LSD's). Through high throughput screening, candidate compounds for metachromatic leukodystrophy will be identified.
- Benefit: The cell-based assay will also indentify small molecules that could have broader application for treatment of other LSD's.
Doug Martin, PhD (Auburn University): Sheep as a Model of Tay-Sachs Disease
- Description: Characterization of disease progression in Tay-Sachs disease sheep and treatment the sheep with AAV vectors).
- Benefit: The sheep is the first true Tay-Sachs disease animal model (as the mouse and cat models are Sandhoff disease models). Also, the large sheep brain is closer in size to the human brain. Therefore, the current proposal is an essential step to test promising therapeutic strategies.
Tay-Sachs Gene Therapy (TSGT) Consortium Update
Since its formation in the summer of 2007, the Tay-Sachs Gene Therapy (TSGT) Consortium has been diligently working toward the goal of starting a clinical trial for Tay-Sachs and Sandhoff in the next several years.
Currently the consortium is working to answer these (very simplified) questions:
- In mice, what is the most effective combination of Tay-Sachs and/or Sandhoff genes in their natural configuration or modified to increase the enzyme production?
- In mice and cats, which type of AAV sub-type is most effective? Based on results in the mice and cat models, the consortium expects to conclude which type is the most efficient AAV particle by mid summer 2008. If it works well in mice and cats, it is reasonable to conclude that it may also be effective in humans.
The Consortium is pleased to share these (very simplified) results:
- It is essential to inject mice early for maximum benefit. Affected mice treated at 1 month are alive and in good health at 18 months; the humane endpoint of untreated affected mice is 4 months. The Consortium anticipates these mice will reach 24 months of age, which is the normal life span of mice. Mice treated at later stages showed some benefit but not as remarkable as the mice treated early.
- Two treated affected cats lived to 7 and 8 months of age; normal lifespan for untreated affected cats is 4.5 months.
- The detailed comparison of the lipids (fats) in the brains of affected mice, cats and humans is nearly complete. The Consortium is also measuring the GM2-ganglioside levels in treated affected cats, and characterizing the changes in myelin (white matter) in mice and cats.
Tay-Sachs & Sandhoff Natural History Update
Looking ahead to the clinical trial, the consortium recognized the need to accurately characterize Tay-Sachs and Sandhoff from the earliest symptoms to end-stages to determine if this (or any therapy) is having a beneficial effect. To develop this natural history, TSGT Consortium researchers, in collaboration with scientists and physicians outside the TSGT Consortium, have developed detailed questionnaires for the three forms of Tay-Sachs and Sandhoff.
The questionnaires were presented to families during the Medical & Scientific Update Session at the 30th Annual Family Conference in Tampa, FL in March 2008. The feed-back was greatly appreciated and was incorporated into the questionnaires during a meeting of clinicians at the Harvard Faculty Club on April 17th.
We need help to make the natural history study as accurate and complete as possible. If you are not currently a member of the PSG of NTSAD and you have or had a loved one affected by Tay-Sachs or Sandhoff, please contact Kim Crawford, Director of Member Services at Kim@ntsad.org or 800-906-8723 to be part of the study. Please note that participation is completely voluntary and will not directly benefit you or your loved one.
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Research Update - March 2010
The Tay-Sachs Gene Therapy Consortium (TSGT), which was awarded an NIH four year $3.5 million grant in August 2009, has issued a research progress report for public release. This marks the beginning of some meaningful progress with a large animal model.
As background, to move an experimental therapy from the lab to the bedside, researchers must show success in a small animal model and a large animal model. There are also toxicity studies and other hurdles to clear before the FDA will approve a human clinical trial. A working large animal model is a hurdle Tay-Sachs therapies have not been able to clear. For the therapy, the large animal model is to treat a naturally born affected cat. Additionally, there is a herd of Jacobs sheep (another potential large animal model) naturally afflicted with Tay-Sachs disease - but preparing sheep for clinical use was thought to be six to twelve months away. There is great news on that front, as well. The following are some of the research highlights.
There are two kinds of cats in the research - GM1 and GM2 - both are lysosomal storage diseases. The GM1 cats are more plentiful, but the GM2 cats are the better Sandhoff/Tay-Sachs model. It is expected that both conditions can be helped by the gene therapy treatment.
- In September 2009 a GM1 cat was born and treated with the gene therapy. Today, six and a half months later, a veterinary neurologist sees NO signs of GM1 disease!! An untreated GM1 cat normally begins showing signs at three and a half to four months. A number of treated GM1 cats are currently in the pipeline. This is VERY encouraging!!
- A GM2 cat was born and treated in January 2010. At two months of age the cat continues to do very well. It is still too early to say the treatment is a success - but at two to two and a half months an untreated cat normally displays whole body tremors - this cat does not. More will be known in four months, but again this progress is very encouraging. Another GM2 cat was treated in late March 2010 - so there are now two treated cats to study.
- As the research team awaits additional GM2 births, they have injected gene therapy vectors into a carrier cat (one good gene and one mutated gene). One month after injection the enzyme levels in the brain and central nervous system were 60-fold higher!! If you took a Tay-Sachs child with a 2% Hex A level and increased it to 4% - it would translate into years more life. If it went up 60 times - that would stop the disease progression altogether!! Increased enzyme expression in the central nervous system is also VERY encouraging.
- The Tay-Sachs sheep colony has produced four affected lambs that are candidates for gene therapy. The research team is creating a sheep vector model and hopes to treat these animals very soon - well ahead of the original plan.
This is very exciting (and very preliminary) research data. We have what appears to be a working large animal model and now an additional large animal model is research ready, well ahead of projections. The next three to six months will be critical for the pioneering cats!! It is still possible the treatment impact will fade or the disease is just progressing slowly in the treated cats. But, today - we have hope!!
Even more good news, the NTSAD is in the final phase of review for research proposals seeking monies. There are a number of very exciting proposals being reviewed by the Scientific Advisory Committee and the Research Initiative team; including a highly scored stem cell project and a sheep research project.
The PYR trials are underway with mixed results. The research team is currently studying the data and working on effective dosage levels and planning the next course of action.
Click here for official progress report from the TSGT Consortium. You can also learn more about the TSGT at http://www.tsgtconsortium.com.
Real progress is being made - the CURE is within reach.
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NTSAD Research Initiative
With its inception in 1957, the founding families envisioned that NTSAD
was to be an umbrella organization that would include not only the more
prominent known disorders at the time: Tay-Sachs, a lysosomal storage
disorder and Canavan, a leukodystrophy, but also related conditions posing
similar clinical symptoms as well as shared familial, emotional and scientific
problems. Fifty years ago, many of these “allied diseases” were
not yet identified or named, but today the “allied diseases” represent
a family of more than 50 genetic diseases including leukodystrophies
and other lysosomal storage diseases affecting the central nervous system.
By supporting scientific research, these families wanted to find the
answers to these questions:
- What causes these diseases?
- How do these diseases progress?
- Can we stop the progression?
- Can we treat the diseases? Can we cure these diseases?
- Can we prevent the diseases?
NTSAD’s support of the scientific process eventually led to some
of the most exciting discoveries and firsts in genetics, including finding
some of the genetic causes of disease and the resultant understanding
of prevention through carrier testing, community screenings, and prenatal
diagnosis. But significant questions about disease progression,
treatment and cure remain.
NTSAD’s Research Initiative strives to:
- clearly communicate disease-related research developments
- provide networking for basic and clinical scientists focused on our
disorders
and funding organizations
- galvanize the lysosomal storage and leukodystrophy diseases community.
NTSAD seeks to define the relevant research by understanding and communicating
research already underway, by promoting the viability of current research,
and by securing public and private sources of research funding.
The Research Initiative Committee is a network of committed and passionate
people directly affected by lysosomal storage and leukodystrophy diseases
dedicated to raising the funds necessary to vigorously pursue treatments
and ultimately cure these devastating diseases.
NTSAD established the Scientific Advisory Research Evaluation Subcommittee
to liaison to the scientific community in order to assess current research
developments and advise the Research Initiative Committee on the leading
scientific efforts. Together these committees endeavor to unite and motivate
scientific and philanthropic leaders from around the world.
NTSAD solicits proposals for innovative research projects that should
generate strong preliminary data for major funding in the area of genetic
neurodegenerative disorders and, in partnership with other organizations
or as part of a larger consortium, the Research Initiative funds research
that can lead to novel treatments for lysosomal or leukodystrophy diseases
impacting the central nervous system.
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Adopt A Sheep Project
by Justin Ungerleider
I have undertaken to raise money to support Tay-Sachs Research, in memory of my brother, Evan Lee Ungerleider and all those who have lost their battle with Tay-Sachs disease. Please help to make a difference in the quest to find a treatment and cure for Tay-Sachs, so that future children and families are not left to suffer.
It has recently been discovered that Tay-Sachs Disease is present in the population of a rare breed of sheep called Jacob Sheep. Amazingly these animals’ genetic make-up is very similar to humans in the way that matters most for solving the mystery of Tay-Sachs Disease. We have to raise funds to help care for the Jacob Sheep who will be the subject of the research to find a treatment and cure for Tay-Sachs. The cost of caring for one sheep is $125 per year. You can help to feed a sheep for a whole year or part of it. The sheep will soon be transported from Texas to New England where the research will be carried out. If you decide to support this project you will receive a certificate with the name of your sheep and its picture. Your support will help to maintain those sheep in the flock that will become part of the research project being developed by the Tay-Sachs Gene Therapy (TSGT) Consortium.
I can be reached at ungy09@yahoo.com for more information.
To “Adopt-a-Sheep” donate online at http://ntsad-ny.org/donations.html or return the form below.
Click here for a printable version.
________________________________________________________________________
____Yes, I would like to Adopt-a-Sheep. Enclosed is my donation of
____$125 to feed one sheep for a year
____$62.50 to feed one sheep for 6 months
____$31.25 to feed one sheep for 3 months
____I do not want to Adopt-a-Sheep, but please accept my donation of $_________.
Print Name__________________________________________
Company____________________________________________
Address_____________________________________________
City/State/Zip________________________________________
Tele.(Day)________________ (Eve.)_____________________
email_______________________________________________
Does your company participate in a matching gift program?____ Yes____ No (If yes, please enclose form.)
Enclosed is my check payable to NTSAD for $__________
If you prefer, we accept __VISA__MASTERCARD__AMEX
CARD NUMBER__________________________EXP. DATE_________
SIGNATURE_________________________________________________
Mail donation to: NTSAD/ELUF
1202 Lexington Avenue #288
New York, NY 10028
All donations are tax deductible to the full extent of the law. A copy of the latest annual report may be obtained, upon request, from our organization, or the Office of the Attorney General, Charities Bureau, 120 Broadway, New York, NY 10271.
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The Odd Connection Between Sheep and Tay-Sachs Disease
by Hanna Bandes Geshelin
What’s the relationship between a Catholic couple with a small Texas acreage, strange spotted sheep named after a biblical story, the administrative assistant of the Rabbinic Association of Greater Dallas (RAGD), and a bunch of east-coast scientists–and why should you care? We are all bound up together in the effort to find a treatment and cure for Tay-Sachs.
A week after I began working as administrative assistant of RAGD, I got a strange message about sheep, a farmer outside of Dallas, and Tay-Sachs disease. The Jewish Federation couldn’t help this man, the operator said; perhaps the Rabbinic Association could? The Rabbinic Association could do nothing. But as an Orthodox Jew I’m familiar with the story of Jacob and the spotted sheep, as a hand spinner I am interested in sheep and wool, and when I was young I had friends who lost three children to Tay-Sachs. Did I say I’m also a writer? I decided to investigate.
I called the farmer, Fred Horak, whose St. Jude’s Farm is named after the patron saint of lost causes. The Horaks raise Jacob Sheep, a nearly extinct breed of unique spotted sheep. He told me that the sheep might hold the treatment and cure to Tay-Sachs disease, but $3000 was needed to transport the sheep to researchers in Massachusetts. As I verified his story, I learned the
following:
Ten years ago a couple of the Horaks’ lambs began to totter and lose their mobility. Then the lambs died. When their veterinarian was mystified, the Horaks, who wanted to get rid of the carriers of this disease, brought the problem to vets at the veterinary medicine school at Texas A & M University (TAMU). Instead of helping the Horaks identify carriers, a researcher
convinced them to breed for this strange disease so that TAMU researchers would have more animals to study.
They began breeding the sheep that they thought might be carriers, as well as raising healthy sheep. Soon the Horaks had about 60 sheep, of which one-third were in the disease-carrying pool. Although the Horaks continued to maintain the carrier sheep at considerable expense, researchers at TAMU seemed to forget about the sheep. Years went by without anyone from TAMU contacting the Horaks. Finally the Horaks decided to butcher some of these sheep; they couldn’t afford to keep them forever.
Then, late last winter Dr. Brian Porter of TAMU called the Horaks saying that TAMU researchers had identified the problem. In June 2008 he went out to the farm, drew blood from all the sheep, and sent it to researchers in New York City who were interested in the problem. Soon the researchers sent back the results: there were eight carriers- six ewes and two rams. The problem was Tay-Sachs disease.
Tay-Sachs has been identified in other animals, most curiously in penguins and flamingos in a few American zoos. But these animals’ genetic make-up is only remotely related to that of humans. These unusual sheep are very similar to humans in the way that matters most for unraveling the mystery of Tay-Sachs disease.
Researchers in the Tay-Sachs Gene Therapy Consortium have put together a plan to use the sheep to find a treatment and cure for the disease. When the Horaks contacted me a few months ago, their problem was money to transport the sheep from Texas to New England, where the research will be carried out. By now the transport fees are covered. However, this initiative, which holds out an excellent chance of finding a treatment and cure for this 100% fatal disease, needs to raise considerably more money. Doctors estimate that between the cost of raising the sheep, maintaining the sheep, and the research itself, they will need $500,000-$1 million. The researchers have already found cures for similar lysosomal storage diseases. They are confident that these sheep hold the key to a cure for Tay-Sachs.
The research plan has come just in time. Some of the ewes are old and have never been bred. Whether they will be able to breed is questionable. One of the rams has never successfully impregnated a ewe. The gene pool is shrinking. This year could be the last year to begin breeding the trait successfully.
To me, everything about this story has been amazing. If you believe in coincidence, that things happen randomly and occasionally fortuitously, then this saga shows an amazing series of coincidences. If you don’t believe in coincidence, then the whole ten-year saga of the Jacob Sheep shows evidence of divine providence.
Now it is our turn to play a role, and in spite of difficult financial times we need to open our wallets and support this effort. There is no time to lose.
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What Are jacob sheep?
 Jacob Sheep are a very ancient breed that probably originated in Syria some 3000 years ago. Pictorial evidence traces the breed's movement through North Africa, Sicily, Spain, and on to England. Jacob sheep were imported into the U.S. for game parks and zoos around the turn of the century. Additional imports from Britain in the 1950s and 60s enhanced the genetic pool, at the time the breed was dwindling. Active preservation efforts saved what was left of the breed and established a healthy genetic pool which assures the breed's survival.
 The Jacob is a small to medium size breed. Adult ewes range from 80 to 140 pounds, while rams will occasionally obtain weights approaching 200 pounds. Coloring is basically white with black or lilac spots randomly distributed on the body. At least 15% of each color must be present. The fleeces from Jacobs are a delight for hand-spinners and for the connoisseur of natural color. Due to the spotting of these animals, the wool can be spun into a complete spectrum from white through gray/lilac to black. This general information about Jacob sheep was provided by the Jacob Sheep Breeders Association.
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NIH Awards $3.5 Million Dollar 4-Year Grant to Tay-Sachs Research Team
The Tay-Sachs Gene Therapy (TSGT) Consortium has received official notification from the National Institutes of Health (NIH) - more percisely the National Institute of Neurological Disorders and Stroke - that it has been awarded a 4 year, $3,545,985 research grant!!! The grant period begins September 1, 2009 with a first year distribution of $1,054,908!!!
The Tay-Sachs Gene Therapy Consortium consists of research experts from Massachusetts General Hospital, Auburn University, Boston College, NYU, University of Massachusetts and Cambridge University in England. They are using gene therapy applications to try to halt the progressive neurological degeneration of Tay-Sachs disease. The first year of research produced unbelievable success in small animal models and vector distribution throughout the brain. The second year of research focused on large animal models. There are naturally occuring models in both cat and sheep populations. In year 3 of 4 (depending on success with large animal models) the Consortium will be preparing a clinical trial protocol and seek approval from regulatory agencies in the US (FDA) and UK.
This is a huge step in our battle to find a cure for Tay-Sachs disease, made possible by the many generous donors that have supported us!!! We are blessed to have such a talented group of researchers working on our cause!!! Prior to obtaining this NIH grant, NTSAD’s Research Initiative awarded $577,000 in grant support in the last two years to the Tay-Sachs Gene Therapy Consortium. These funds were critical in advancing the project and enabling the scientific team to apply for the NIH funds. Thousands of people financed this effort through the Cure Tay-Sachs Foundation (CTSF), Cameron & Hayden Lord Foundation, NTSAD New York Chapter, Harry Hoffman Fund, Evan Lee Ungerleider Foundation, Mathew Forbes Romer Foundation and the Sophia Pesotchinsky Fund. This is a huge step in our battle to find treatments and cures for Tay-Sachs disease!
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