AAV2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial.
T. Aleman; Rachel M. Huckfeldt; Leona W. Serrano; Denise J. Pearson; Grace K. Vergilio; Sarah Mccague; Kathleen A. Marshall; M. Ashtari · 2022 · Ophthalmology (Rochester, Minn.)
WASTE classifies this as Negative / Null Result Report · AI classification, approximate
The study found no significant effect — useful as a negative control or null benchmark for your own design.
Abstract (excerpt)
PURPOSE To assess the safety of the subretinal delivery of a recombinant adeno-associated virus serotype 2 (AAV2) vector carrying a human CHM-encoding cDNA in choroideremia (CHM). DESIGN Prospective, open-label, non-randomized,…
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Metadata source: Semantic Scholar · DOI 10.1016/j.ophtha.2022.06.006