Publication date: Apr 06, 2019
CHICAGO, April 5, 2019 /PRNewswire/ — Scientist from Dystrogen Therapeutics Corp. will present data supporting a potential cutting-edge therapy for neurodegenerative diseases caused by trinucleotide repeats, such as Huntington’s disease (HD) and spinocerebellar ataxias (SCAs).
“These findings are potentially significant for the treatment of Huntington’s disease and SCA patients, and the ability to selectively silence CAG transcripts in the nucleus may prove to be critical for therapeutic efficacy of gene therapies for these diseases,” stated Kris Siemionow, M. D., Ph. D., chief executive officer of Dystrogen.
The company has two technology platforms, which focus on treating patients with rare diseases such as Duchenne muscular dystrophy, sickle cell anemia, and neurodegenerative disorders such as Huntington’s disease and SCA.
Dystrophin expressing chimeras “DEC” are based on ex vivo fusion of allogeneic human myoblast derived from close relative donors with autologous human myoblast received from DMD patient, where chimeric cells maintain the ability to express normal dystrophin protein.
The company is planning on enrolling patients for its DEC chimeric cell therapy Duchenne muscular dystrophy trial.
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- Prevalence of Carriers of Intermediate and Pathological Polyglutamine Disease-Associated Alleles Among Large Population-Based Cohorts.
- Huntingtin Lowering Strategies for Disease Modification in Huntington’s Disease.
- Combined Stem Cell, Gene Therapies Ease Symptoms, Prolong Survival in HD Mouse Model