Major Improvement in CRISPR Technology Could Fix Genetic Spelling Mistakes: Implications for Neuromuscular Disorders?
CRISPR-Cas9 technology, a gene editing tool, has been significantly upgraded with a new feature called prime editing. This enhancement addresses two limitations of CRISPR technology by: (1) allowing the change of a gene’s spelling into a predicted sequence without requiring a cell to divide and (2) without needing to cut both strands of the DNA double helix, which reduces the risk of making unintentional changes. The study published in Nature used prime editing on human cells to correct the genetic spelling mistakes associated with Tay-Sacks and Sickle-Cell Anemia. Compared to traditional CRISPR technology, this study showed that the prime editing feature made CRISPR more precise, efficient and highly versatile.
CRISPR-Prime editing holds promise for the treatment of neuromuscular disorders (NMDs) as it allows genetic repairs in non-dividing cells like neurons and muscle cells and could potentially be used on a subset of NMDs caused by small genetic spelling mistakes like point mutations, small insertions or deletions. But in its current form, up to 80 letter changes were edited and so this upgrade would not work for other types of NMDs: Charcot-Marie-Tooth 1A and Myotonic muscular dystrophies as well as some cases of Duchenne, and Becker Muscular Dystrophies are caused by large thousand or even hundred thousand letter deletions, duplications, inversions and repeat expansions. As this technique moves us one step closer to correcting genetic mutations that cause human genetic diseases, there is a need to continue research to develop safe and effective treatment for the wide range of specific disease-causing spelling mistakes associated with NMDs
Alberta Fourth Province to Expand Access to Spinraza™ for Patients Impacted with Spinal Muscular Atrophy
Alberta fourth province to expand access to SPINRAZA™ for patients impacted with Spinal Muscular Atrophy.
Muscular Dystrophy Canada (MDC) commends the Government of Alberta for joining Quebec, Saskatchewan, and Ontario in expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA).
In Alberta, the following patients will now be eligible for reimbursement of SPINRAZA™, in addition to Type 1 patients:
- patients who are pre-symptomatic with two or three copies of SMN2, or
- have had disease duration of less than six months, two copies of SMN2, and symptom onset the first week after birth and on or before seven months of age, or
- are under the age of 18 with symptom onset after six months of age, regardless of the ability to walk.
- Other patients who do not meet the expanded funding criteria may be considered in exceptional cases.
More Patients Impacted with Spinal Muscular Atrophy in Ontario to Gain Access to Spinraza™
Toronto, Ontario – Muscular Dystrophy Canada (MDC) applauds the Government of Ontario for expanding access to SPINRAZA™, a life-changing treatment for individuals impacted with Spinal Muscular Atrophy (SMA).
In Ontario, expanded coverage of SPINRAZA™ will include the following, in addition to existing coverage for Type 1 patients:
- patients who are pre-symptomatic with two or three copies of the SMN2 gene;
- patients with a disease duration of less than six months, two copies of the SMN2 gene, and symptom onset the first week after birth and on or before seven months of age;
- patients under the age of 18, with symptom onset after six months of age and who have never achieved the ability to walk independently.
MDC presents CADTH With GMG Patient Submission
MDC pleased to provide CADTH with a patient submission from our generalized Myasthenia Gravis (GMG) community in support of Soliris.
MDC remains committed to ensuring that neuromuscular clients have access to the latest
treatments that offer positive health outcomes.
Download the patient submission
ABOUT MUSCULAR DYSTROPHY CANADA
Muscular Dystrophy Canada’s mission is to enhance the lives of those impacted with neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for a cure through well-funded research. To learn more about Muscular Dystrophy Canada, please visit muscle.ca or call our toll-free number at 1-800-567-2873.
MEDIA CONTACT
Heather RiceMuscular Dystrophy Canada
Heather.Rice@muscle.ca
Treating Duchenne Muscular Dystrophy Focus of New Research Partnership
For Immediate Release – April 4, 2019
Toronto, Ontario – Muscular Dystrophy Canada (MDC) and The Foundation for Gene & Cell Therapy (Jesse’s Journey) are joining forces to accelerate ground-breaking research focused on new treatments for Duchenne muscular dystrophy to the sum of $600,000.
Through this partnership, MDC will provide $300,000 with Jesse’s Journey matching the commitment. Two research projects will be funded:
- Dr. Anthony Gramolini: Non-viral, immune-modulatory nanoparticles for delivery of CRISPR/Cas9 as a treatment intervention for Duchenne Muscular Dystrophy.
- Dr. Michael A. Rudnicki: Exosomal Delivery of Wnt7a for treating Duchenne Muscular Dystrophy.
“For anyone impacted by a neuromuscular disorder, research offers hope that better treatments will be uncovered, and ultimately, a cure. Both of these research projects show very promising initial results and we are thrilled that this investment will help move the research forward,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We are thrilled to be working with Jesse’s Journey to fund this promising research and can’t wait to see what positive impacts it has on our clients and families.”
Barbara Stead-Coyle
CEO, Muscular Dystrophy Canada
Federal Government Commits to National Strategy to Improve Access to Rare Disease Drugs
Toronto, Ontario – Muscular Dystrophy Canada commends the Government of Canada for committing $35 million, in its 2019 budget, to develop a Canadian Drug Agency to oversee the development of a new national formulary of prescribed drugs and a strategy to provide support for Canadians with rare diseases.
A national strategy to alleviate the high-cost of drugs for rare diseases will ensure more Canadians are able to access effective, life-changing treatments.
“This is an important first-step towards ensuring individuals diagnosed with a rare disease, such as a neuromuscular disorder, are able to access treatment without cost as a barrier,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We call on all Federal parties to focus on this very real issue facing Canadians.”
CADTH REVIEW AND SPINRAZA
Spinraza decision signals the need for a Canadian Rare Disease (or Orphan Drug) Framework
The drug review and approval process in Canada is complex. Many agencies from Health Canada, to Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et en services sociaux (INESSS), to the Pan-Canadian Pharmaceutical Alliance and each individual provincial and territorial governments all share in the decision-making.
MDC Welcomes Daria Wojtal as Director of Research
We are pleased to announce that Daria Wojtal has joined Muscular Dystrophy Canada as the new Director of Research. Daria comes to us from the Hospital for Sick Children and the University of Toronto, where she worked under the leadership of Dr. Ronald Cohn on her PhD in developing CRISPR gene editing technology for the treatment of neuromuscular disorders.