Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington’s Disease

Dystrogen Therapeutics Announces That Gene Therapy Successfully Cuts Off Production of Neuron-Destroying Protein in Huntington’s Disease

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.

Concepts Keywords
Allogeneic Inflammation
Anemia Rare genetic diseases
Autologous Immunosuppression
CHICAGO Branches of biology
Chief Executive Officer Organ systems
Chimeras Neurological disorders
Chimeric Muscular dystrophy
Congress RTT
DEC Gene expression
DMD RNA
Duchenne Muscular Dystrophy Huntington’s disease
Dystrophin Duchenne muscular dystrophy
Fibrotic Spinocerebellar ataxia
Genetic Gene silencing
Genetic Diseases Dystrophin
Huntingtin
Huntingtin Protein
Huntington
Immune System
Immunosuppression
Inflammation
Mortality
Muscle
Mutant
Myoblast
Myoblasts
Neurodegenerative
Neurodegenerative Diseases
Neurodegenerative Disorders
Neuron
Nucleus
Pediatric
PolyQ
Protein
RNAi
Sickle
Spinocerebellar Ataxias
Toxicity
Trinucleotide Repeats
Vivo

Semantics

Type Source Name
pathway BSID Immune System
disease MESH inflammation
gene UNIPROT PTEN
gene UNIPROT DMD
disease DOID sickle cell anemia
disease MESH sickle cell anemia
disease DOID Duchenne muscular dystrophy
disease MESH Duchenne muscular dystrophy
disease MESH rare diseases
disease MESH genetic diseases
disease MESH development
disease MESH spinocerebellar ataxias
pathway BSID Neurodegenerative Diseases
disease MESH neurodegenerative diseases
gene UNIPROT TNFSF13
gene UNIPROT ANP32B

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