Choroideremia: the endpoint endgame
MEA Abdalla Elsayed, LJ Taylor, AS Josan… - International Journal of …, 2023 - mdpi.com
… Choroideremia is an X-linked retinal degeneration resulting … from clinical trials in
choroideremia, review endpoints, and … be used as endpoints must be considered for …
choroideremia, review endpoints, and … be used as endpoints must be considered for …
Choroideremia: The Endpoint Endgame
L Taylor, A Josan, M Fischer… - International Journal of …, 2023 - europepmc.org
… Choroideremia is an X-linked retinal degeneration resulting … from clinical trials in
choroideremia, review endpoints, and … be used as endpoints must be considered for …
choroideremia, review endpoints, and … be used as endpoints must be considered for …
Gene Augmentation of CHM Using Non-Viral Episomal Vectors in Models of Choroideremia
L Toualbi, M Toms, PV Almeida, R Harbottle… - International Journal of …, 2023 - mdpi.com
… promising treatment options for choroideremia and other IRDs… Choroideremia is a good
candidate for gene augmentation … the primary endpoints and key secondary endpoints after 12 …
candidate for gene augmentation … the primary endpoints and key secondary endpoints after 12 …
Gene therapy for inherited retinal diseases: progress and possibilities
ML Hu, TL Edwards, F O'Hare, DG Hickey… - Clinical and …, 2021 - Taylor & Francis
… Choroideremia is an X-linked IRD caused by mutations in the CHM gene. Choroideremia …
have indicated a failure to meet primary endpoints, as improvements from baseline best …
have indicated a failure to meet primary endpoints, as improvements from baseline best …
[PDF][PDF] Gene Therapy Updates for Inherited Retinal Dystrophies
CX Qian - researchgate.net
… The most prevalent example, choroideremia (CHM), will be discussed in this review. …
Choroideremia is caused by a mutation in the CHM gene, which encodes the Rab escort protein 1 (…
Choroideremia is caused by a mutation in the CHM gene, which encodes the Rab escort protein 1 (…
Efficacy of adeno-associated virus serotype 9-mediated gene therapy for AB-variant GM2 Gangliosidosis
M Vyas, NM Deschenes, KJL Osmon, Z Chen… - International journal of …, 2023 - mdpi.com
… term (60-week endpoint) (Figure 4). … endpoint, vector copies in the brain were substantially
higher in the Gm2a −/− mice treated at 6-weeks of age than they were at the 20-week endpoint…
higher in the Gm2a −/− mice treated at 6-weeks of age than they were at the 20-week endpoint…
Contiguous Gene Syndromes and Hearing Loss: A Clinical Report of Xq21 Deletion and Comprehensive Literature Review
MT Bonati, A Feresin, P Prontera, P Michieletto… - Genes, 2024 - mdpi.com
… Although some authors suggested an age-dependent penetrance for choroideremia [17],
in our patient, an early onset of the phenotype has been documented. As reported for females …
in our patient, an early onset of the phenotype has been documented. As reported for females …
[PDF][PDF] Gene therapy for inherited retinal diseases: progress and possibilities
ZL Wang, LN Ayton - 2021 - researchgate.net
… Choroideremia primarily affects the RPE with secondary … a failure to meet primary endpoints,
as improvements from baseline … Endgame: glybera finally recommended for approval as the …
as improvements from baseline … Endgame: glybera finally recommended for approval as the …
A review of CRISPR tools for treating usher syndrome: applicability, safety, efficiency, and in vivo delivery
L Major, ME McClements, RE MacLaren - International Journal of …, 2023 - mdpi.com
This review considers research into the treatment of Usher syndrome, a deaf-blindness
syndrome inherited in an autosomal recessive manner. Usher syndrome mutations are markedly …
syndrome inherited in an autosomal recessive manner. Usher syndrome mutations are markedly …
Bioengineering strategies for restoring vision
… a novel and clinically meaningful trial endpoint (instead of the … ); and second, the endpoint
involved an improvement in visual … g–i, In choroideremia, there is a classic fundus appearance …
involved an improvement in visual … g–i, In choroideremia, there is a classic fundus appearance …