Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Sign Up to like & getrecommendations! Published in 2023 at "Annals of clinical and translational neurology"
DOI: 10.1002/acn3.51772
Abstract: OBJECTIVE Mucopolysaccharidosis type IIIA (MPSIIIA) caused by recessive SGSH variants results in sulfamidase deficiency, leading to neurocognitive decline and death. No disease-modifying therapy is available. The AAVance gene therapy trial investigates AAVrh.10 overexpressing human sulfamidase… read more here.
Keywords: intracerebral gene; mucopolysaccharidosis; focal lesions; therapy ... See more keywords
Intracerebral gene therapy in 4 children with Sanfilippo B syndrome : 5.5 years follow-up results.
Sign Up to like & getrecommendations! Published in 2021 at "Human gene therapy"
DOI: 10.1089/hum.2021.135
Abstract: We report safety (primary endpoint) and efficacy (secondary endpoint) of novel intracerebral gene therapy at 5.5 years of follow-up in children with Sanfilippo B. An uncontrolled phase 1/2 clinical trial was performed in 4 patients… read more here.
Keywords: children sanfilippo; years follow; gene therapy; intracerebral gene ... See more keywords