gene therapy

gene therapy research

New Cell Lines Useful for Screening Gene Therapies for Pompe Disease

Spanish researchers generated different isogenic mouse muscle cell lines that mimic severe Pompe disease and validated their applicability as preclinical models of the disease. These cell lines can be used to screen potential gene therapy strategies for the disease while reducing the number of animals used in research. The generated cell lines lack the acid…

DNA gel close-up

Prime Editing Could Be Used to Correct DMD-Causing Point Mutations, Study Says

Prime editing allows the introduction of specific mutations in the DMD gene, a new study published in the International Journal of Molecular Sciences reported.  Based on this finding, the authors of the study concluded that prime editing could therefore be used to correct point mutations in the DMD gene causing Duchenne muscular dystrophy (DMD).  For…

Chromosome DNA

Novel CRISPR/Cas System May Efficiently Repair Mutations Causing DMD and CF

A novel clustered regularly interspaced short palindromic repeats (CRISPR)/Cas-based technology developed by Script Biosciences can correct mutations causing Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF) with high efficiency and low on-target DNA damage rate, according to a study presented at the 25th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting. According to…

Gene therapy concept

AAVs Appear to Be Effective Vectors for Gene Therapy in LCFAOD

Gene therapy using an adeno-associated virus (AAV) appears to amend important abnormalities in patients with long chain fatty acid oxidation disorder (LCFAOD), according to an article recently published in Molecular Therapy. Specific genetic therapy for very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency, a form of LCFAOD, with AAV resulted in improvement of laboratory test results, evident…

gene therapy

New Gene Therapy Approach Holds Potential Therapeutic Value in LAL-D

Researchers from France developed a gene therapy approach based on hematopoietic stem cells for the erythroid-specific expression of lysosomal acid lipase (LAL) enzyme in the blood as a potential curative treatment for LAL deficiency (LAL-D). The LAL enzyme is essential for the hydrolysis of cholesteryl ester and triglyceride in the lysosome. So its deficiency leads…

gene therapy

Engineered Enzyme Variants May Have Better Efficacy in Pompe Disease Therapy

New enzyme variants developed by Codexis may have better efficacy than enzymes currently used for enzyme replacement therapy in patients with Pompe disease and other diseases, according to preclinical data presented at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting.  The engineered acid alpha-glucosidase (GAA) enzyme variants for Pompe disease retained…

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