Gene Therapy Research Review, Issue 5
In this issue:
First use of topical B-VEC for ocular complications of DEB
In vivo CRISPR gene editing promising for inherited retinal blindness
Enhancing clinical infrastructure for the delivery of genetic therapies
Once off ixo-vec may replace regular anti-VEGF injections for wet AMD
Descartes-08 elicits long-term improvements in myasthenia gravis
Stem cell-derived dopaminergic grafts feasible for Parkinson’s disease
Natural killer cell therapy with logic-gated gene circuits for AML
Exa-cel functionally cures sickle cell disease
Exa-cel reduces transfusion dependence in β-thalassemia
Intrathecal gene therapy may slow progression of giant axonal neuropathy
RGX-202 under investigation for Duchenne muscular dystrophy
Expert contributor
Dr Heidi Peters
Dr Heidi Peters is a biochemical geneticist who works as a specialist in the metabolic unit at Genetic Health Services Victoria. She also heads research aimed at developing novel therapies for inborn errors of metabolism.
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