The DTX101 program was halted for insufficient treatment response; however, from its completion, lessons can be learned regarding the design and execution of gene therapy clinical trials, including additional optimization of transgene sequence and immunosuppression protocols. The 101HEMB01 and 101HEMB02 ...
In conclusion, our data demonstrate that ACE2 gene therapy could have a potential therapeutic value in T2DM by attenuating pancreatic fibrosis.doi:10.1096/fasebj.27.1_supplement.1154.7Harshita ChodavarapuPharmacology and Experimental TherapeuticsLouisiana State UniversityNew OrleansLAKavaljit Chhabra...
Richard Mulligan with the objective of promoting the use of gene therapy and to conduct research developing new gene delivery vector technologies. Iowa Center for Gene Therapy, University of Iowa, Iowa City Diseases of the lung, cardiovascular system, muscles, brain, and skin are focus areas of...
Pompe disease is a neuromuscular disease caused by a deficiency of the lysosomal enzyme acid alpha-glucosidase leading to lysosomal and cytoplasmic glycogen accumulation in neurons and striated muscle. In the decade since availability of first-generation enzyme replacement therapy (ERT) a better understan...
Central Illustration: Mechanisms of gene therapy toxicity. TLR: toll-like receptor. PAMP: pathogen-associated molecular patterns, APC: antigen-presenting cell, NF-kB: nuclear factor kappa B, ITR: inverted terminal repeats, pA: poly-A tail. Created with BioRender.com Download: Download high-res ...
financial interest in the use of rAAV therapies and own equity in Applied Genetic Technologies Corp., a company that may commercialize some aspects of this work. The University of Pennsylvania, the University of Florida and Cornell University hold a patent on the...
Some researchers hoping that gene-editing technology can conquer forms of Alzheimer’s caused by genetic mutations. By Tosin Thompson Twitter Facebook Email Last month saw the first-ever approval of a gene therapy that uses the CRISPR–Cas9 gene-editing tool, a treatment for the blood conditions...
An experimental gene therapy restored the night vision of two people with leber congenital amaurosis.When you purchase through links on our site, we may earn an affiliate commission. Here’s how it works. A new gene therapy restored the night vision of people with an inherited eye disorder. ...
Tuschl et al. “Small Interfering RNAs: A Revolutionary Tool for the Analysis of Gene Function and Gene Therapy”, Molecular Interventions, (2002), vol. 2, No. 3, pp. 158-167. Rubinson et al. “A lentivirus-based system to functionally silence genes in primary mammalian cells, stem cells...
AAV has been shown to infect a variety of cell and tissue types, and significant progress has been made over the last decade to adapt this viral system for use in human gene therapy. In its normal “wild type” form, recombinant AAV (rAAV) DNA is packaged into the viral capsid as a ...