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
- Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa® in Duchenne Muscular Dystrophy (DMD) and Focus on Vamoroloneon October 5, 2020 at 10:19 pm
Stock quotes by finanzen.net Santhera will hold a conference call today at 13:00 CEST, 12:00 BST, 07:00 EDT. Details are at the end of this statement. Pratteln, Switzerland, October 6, 2020 – Santhera ...
- Santhera to Discontinue Phase 3 SIDEROS Study and Development of Puldysa® in Duchenne Muscular ...on October 5, 2020 at 10:00 pm
Santhera Pharmaceuticals (SIX: SANN) announces the discontinuation of its Phase 3 SIDEROS study with Puldysa® (idebenone) in patients with Duchenne muscular dystrophy (DMD) who are in respiratory ...
- Drugs controller approves human trials for Duchenne muscular dystrophy drugon October 5, 2020 at 4:36 am
The disease affects only boys, and those who have this condition eventually need a wheelchair because they stop walking by the age of 10 or 12 ...
- FDA Lifts Solid Bio Clinical Hold, Duchenne Study Cleared to Resumeon October 1, 2020 at 9:45 am
Solid Biosciences has answered the FDA’s outstanding questions about its Duchenne muscular gene therapy, clearing a clinical trial to now resume after ...
- Pfizer's Gene Therapy Candidate For Duchenne Muscular Dystrophy Receives Fast Trackon October 1, 2020 at 4:16 am
(PFE) said the company's investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the FDA. The designation ...
Go deeper with Google Headlines on:
Duchenne muscular dystrophy
Go deeper with Bing News on:
Gene correction therapy
- Stem Cell Therapy for Cystic Fibrosis: Current Status and Future Prospectson October 2, 2020 at 5:00 pm
In addition, the duration of gene expression has been ... An approach considering stem cell-based therapy of CF-injured lungs should achieve a permanent correction of the CF genetic defect.
- Gene Editing Market Size Worth USD 15.79 Billion By 2027 | CAGR of 17.2%: Emergen Researchon September 29, 2020 at 6:04 am
The growth of the market is attributed to rising government initiatives across major nations and expanding application areas of genomics - Market Size - USD 4.44 Billion in 2019, Market ...
- Precision BioSciences to Present at Upcoming Virtual Investor Conferences in Octoberon September 28, 2020 at 4:00 am
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology company developing allogeneic CAR T and in vivo gene correction therapies with its ...
- Precision BioSciences and Servier Expand CAR T Oncology Development Collaboration with Four New Programs Targeting Hematological and Solid Tumorson September 24, 2020 at 3:34 pm
Precision BioSciences, Inc., a clinical stage biotechnology developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS ® genome editing platform, in collaboration with Servier, ...
- Precision BioSciences and Servier Expand CAR T Oncology Development Collaboration with Four New Programs Targeting Hematological and Solid Tumorson September 17, 2020 at 5:01 am
17, 2020 (GLOBE NEWSWIRE) -- Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage biotechnology developing allogeneic CAR T and in vivo gene correction therapies with its ARCUS® genome ...