via LMU
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.
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Duchenne muscular dystrophy
- Capricor Therapeutics Announces Follow-up Type-B Clinical Meeting with the FDA for CAP-1002 for the Treatment of Duchenne Muscular Dystrophy
“We are appreciative of the FDA’s guidance and are encouraged by ongoing discussions as we determine the most expeditious path forward for CAP-1002 for the treatment of Duchenne muscular dystrophy (DMD) and align on key features of our Phase 3 HOPE-3 ...
- Reuters Health News Summary
The company was evaluating the efficacy and safety of pamrevlumab in a late-stage trial in patients with non-ambulatory Duchenne muscular dystrophy (DMD), where upper limb function of the patients are affected,
- ‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?
The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.
- Discovery slows down muscular dystrophy
Jan. 31, 2019 — Researchers have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. The team restored muscle stem ...
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Duchenne muscular dystrophy
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Gene correction therapy
- RNA-targeted Splice-correction Therapy for Neuromuscular Disease
Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction ... reading-frame in the DMD gene leading to the absence of ...
- ‘It’s a vote for hope’: first gene therapy for muscular dystrophy nears approval, but will it work?
The FDA’s decision, expected this month, follows several setbacks and delays and will pose difficult choices for the families of children with Duchenne muscular dystrophy.
- First-of-Its-Kind Gene Therapy Can Be Applied to Skin Instead of Injected
Dubbed Vyjuvek, the FDA-approved gene therapy directly delivers healthy copies of a mutated gene onto damaged skin.
- Discovery Slows Down Muscular Dystrophy
A team of researchers at the University of Houston College of Pharmacy is reporting that by manipulating TAK1, a signaling protein that plays an important role in development of the immune system, ...
- Rocket stock rises amid data from multiple gene therapy programs
Fanconi Anemia (FA) The company reported ongoing data from a phase 2 trial its ex vivo lentiviral gene therapy RP-L102 for FA. Rocket said RP-L102, provided sustained genetic correction in 8 of ...
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Gene correction therapy
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