Muscles need dystrophin in order to regenerate. Persons suffering from Duchenne muscular dystrophy lack this essential muscular protein due to mutations in the gene which is responsible for producing dystrophin. As a result, their existing muscle cells deteriorate over time and are gradually replaced by connective and fatty tissue; muscle strength weakens during the course of the disease. The first symptoms usually appear around the age of five. Children with the disease begin to have difficulties with movements they previously completed with ease, for example climbing stairs or getting up from the floor. At approximately the age of twelve, they are no longer able to walk, later losing movement in their arms and hands. Due to concomitant respiratory and cardiac failure, the majority of patients does not reach the age of 40. DMD affects mainly boys, since the responsible mutations are located in the dystrophin gene on the X chromosome.
Gene scissors remove defective gene sequence
An interdisciplinary Munich research team led by scientists from TUM has for the first time succeeded in correcting the mutated dystrophin gene in living pigs. In order to cut the defective gene sequence from the DNA of the animals’ muscle and heart cells, the researchers modified the Crispr-Cas9 gene scissors. “These gene scissors are highly efficient and specifically corrected the dystrophin gene,” says Prof. Wolfgang Wurst, developmental geneticist at TUM and the German Research Center for Environmental Health. It became then again possible to viably read the gene which had been unreadable because of the genetic defect, thus allowing for a successful protein biosynthesis. Now the shorter but stably formed dystrophin protein was able to improve muscle function. The animals treated were less susceptible to cardiac arrhythmia and had an increased life expectancy compared to animals with the disease that did not receive the therapy.
A permanent therapy
“Muscle and heart cells are long-lived cell structures. One half of all myocardial cells remain functional from birth throughout the entire lifecycle of a human being,” says Prof. Christian Kupatt, cardiologist at university hospital TUM Klinikum rechts der Isar. “The genome of a cell is used for protein biosynthesis as long as the cell is alive, and once a cell has been affected by the therapy, it remains corrected. So if we change the genome of a myocardial cell, the correction is a long-term success, in contrast to the results of previous methods.”
Therapeutic success with clinically relevant model
The gene sequence responsible for the dystrophin protein has already been successfully corrected in the past, however in mice and other animal models. “Our results are very promising, since for the first time, we have now been able to demonstrate therapeutic success in a clinically relevant large animal model,” says Prof. Maggie Walter, neurologist at the LMU university hospital. In terms of important biochemical, clinical and pathological changes, the pig model mirrors Duchenne muscular dystrophy in humans. “Since the disease proceeds faster in our pig model, we were able to verify the efficacy of the therapeutic approaches within a manageable period of time,” says Prof. Eckhard Wolf, LMU specialist in veterinary medicine.
The Latest Updates from Bing News & Google News
Go deeper with Bing News on:
Duchenne muscular dystrophy
- Annual Charity Golf Outing ‘Jumping Jack to End Duchenne’ Hosts “The Final Round”
Golf Tournament that Supports Parent Project Muscular Dystrophy Will Celebrate Legacy of Beloved Mother Angela Knight. WESTMINSTER, Colo., June 18, 2022 /PRNewswire/ -- For 19 years, the Knight family ...
- FibroGen Announces Completion of Patient Enrollment in LELANTOS-2, a Phase 3 Clinical Study of Pamrevlumab in Duchenne Muscular Dystrophy
SAN FRANCISCO, June 08, 2022 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced completion of patient enrollment for LELANTOS-2, a Phase 3 clinical study of pamrevlumab in patients with ...
- Satellos Bioscience Co-Founder and Chief Scientific Officer Awarded Prestigious Defeat Duchenne Canada 2022 Grant
the country's only national charity dedicated to ending Duchenne muscular dystrophy ("Duchenne"). "We're very excited for Michael and his academic team at the Ottawa Hospital Research Institute ("OHRI ...
- Defeat Duchenne Canada Grants $1M to DMD Treatment Research
The three research projects Defeat Duchenne Canada is funding brings to $16 million the total awarded in the organization’s 27-year history.
- Defeat Duchenne Canada grants 1.14 million dollars to Duchenne muscular dystrophy research
Thousands of families are in a race against time to find a cure for Duchenne muscular dystrophy - the most common fatal genetic disease diagnosed in childhood. LONDON, ON, June 15, 2022 /CNW/ - Defeat ...
Go deeper with Google Headlines on:
Duchenne muscular dystrophy
Go deeper with Bing News on:
Gene correction therapy
- Global Cancer Gene Therapy Market Report to 2028 - Size, Share & Industry Trends Analysis Report
The "Global Cancer Gene Therapy Market Size, Share & Industry Trends Analysis Report By Therapy (Gene Induced Immunotherapy, Oncolytic Virotherapy, and Gene Transfer), By End User, By Regional Outlook ...
- The Worldwide Cancer Gene Therapy Industry is Expected to Reach $5 Billion by 2028 - ResearchAndMarkets.com
Gene therapy is the process of replacing faulty or damaged genes at the molecular level. It is a procedure in which viruses are genetically modified before being transmitted to ho ...
- OliX Pharmaceuticals Subsidiary mCureX Announces mRNA Technology Collaboration with ToolGen for Rare Eye Disease
OliX Pharmaceuticals, Inc., a leading developer of RNAi therapeutics, today announced that the Company’s subsidiary mCureX Therapeutics, Inc. has recently signed a contract for joint research with ...
- Gene Therapy Market - Growth, Trends, COVID-19 Impact, and Forecasts (2022 - 2027)
The Gene Therapy Market was estimated to be USD 4,118. 31 million in 2021 and is poised to grow at a CAGR of 25. 71% by 2027 to reach USD 14,824. 97 million. The COVID-19 pandemic is expected to have ...
- Severe Immunodeficiencies: Bone Marrow Transplantation or Gene Therapy?
Nonetheless, a partial correction was achieved also in B-cell ... The 2 patients who developed leukemia following gene therapy were the youngest, at 1 and 3 months of age. Further studies are ...