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Current Status of Clinical Trials Design and Outcomes in Retinal Gene Therapy.
Rosin B, Banin E, Sahel JA. Rosin B, et al. Among authors: sahel ja. Cold Spring Harb Perspect Med. 2023 Sep 11:a041301. doi: 10.1101/cshperspect.a041301. Online ahead of print. Cold Spring Harb Perspect Med. 2023. PMID: 37696658
With the rapid expansion of methods encompassed by the term gene therapy, new trials exploring the safety and efficacy of these methods are initiated more frequently. ...
With the rapid expansion of methods encompassed by the term gene therapy, new trials exploring the safety and efficacy of these metho …
Adaptive Optics Flood Illumination Ophthalmoscopy in Nonhuman Primates: Findings in Normal and Short-term Induced Detached Retinae.
Dentel A, Brazhnikova E, Norberg N, Jaillard C, Grieve K, Paques M, Sahel JA, Bertin S, Forster V, Picaud S. Dentel A, et al. Among authors: sahel ja. Ophthalmol Sci. 2023 Apr 20;3(4):100316. doi: 10.1016/j.xops.2023.100316. eCollection 2023 Dec. Ophthalmol Sci. 2023. PMID: 37274010 Free PMC article.
OBJECTIVE: To describe adaptive optics flood illumination ophthalmoscopy (AO-FIO) of the photoreceptor layer in normal nonhuman primates (NHPs) and in the case of a short-term induced retinal detachment (RD). DESIGN: Longitudinal fundamental research study. ...
OBJECTIVE: To describe adaptive optics flood illumination ophthalmoscopy (AO-FIO) of the photoreceptor layer in normal nonhuman primates (NH …
Relentless placoid chorioretinitis: A review of four cases in pediatric and young adult patients with a discussion of therapeutic strategies.
Zaheer HA, Odden J, Gagrani M, Zaguia F, Lowder C, Coca A, Rosenkranz ME, Patil-Chhablani P, Ores R, Boussion F, Indermill C, Sahel JA, Nischal K, Goldstein DA, Errera MH. Zaheer HA, et al. Among authors: sahel ja. Front Pediatr. 2023 Mar 27;11:885230. doi: 10.3389/fped.2023.885230. eCollection 2023. Front Pediatr. 2023. PMID: 37051435 Free PMC article. Review.
After the failure of interferon-alpha-2a, one patient displayed long-term control with infliximab. One patient did not require a steroid-sparing agent after oral prednisone taper as there was no evidence of progression or recurrence. ...
After the failure of interferon-alpha-2a, one patient displayed long-term control with infliximab. One patient did not require a ster …
Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation.
Carelli V, Newman NJ, Yu-Wai-Man P, Biousse V, Moster ML, Subramanian PS, Vignal-Clermont C, Wang AG, Donahue SP, Leroy BP, Sergott RC, Klopstock T, Sadun AA, Rebolleda Fernández G, Chwalisz BK, Banik R, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA; the LHON Study Group. Carelli V, et al. Among authors: sahel ja. Ophthalmol Ther. 2023 Feb;12(1):401-429. doi: 10.1007/s40123-022-00611-x. Epub 2022 Nov 30. Ophthalmol Ther. 2023. PMID: 36449262 Free PMC article.
METHODS: The visual acuity of 174 MT-ND4-carrying patients with LHON injected in one or both eyes with lenadogene nolparvovec from four pooled phase 3 studies (REVERSE, RESCUE and their long-term extension trial RESTORE; and REFLECT trial) was compared to the spontaneous e …
METHODS: The visual acuity of 174 MT-ND4-carrying patients with LHON injected in one or both eyes with lenadogene nolparvovec from four pool …
Long-term observations of macular thickness after subretinal implantation of a photovoltaic prosthesis in patients with atrophic age-related macular degeneration.
Muqit MMK, Mer YL, Holz FG, Sahel JA. Muqit MMK, et al. Among authors: sahel ja. J Neural Eng. 2022 Oct 14;19(5):10.1088/1741-2552/ac9645. doi: 10.1088/1741-2552/ac9645. J Neural Eng. 2022. PMID: 36174540 Free PMC article.
Based on the analysis of three patients with subretinal implantation, we found that the distance between the implant and the target cells was stable over the long-term follow-up. Total RT above the implant decreased on average, by 39 12m during 3 months post-implantation, …
Based on the analysis of three patients with subretinal implantation, we found that the distance between the implant and the target cells wa …
Absence of lenadogene nolparvovec DNA in a brain tumor biopsy from a patient in the REVERSE clinical study, a case report.
Newman NJ, Schniederjan M, Mendoza PR, Calkins DJ, Yu-Wai-Man P, Biousse V, Carelli V, Taiel M, Rugiero F, Singh P, Rogue A, Sahel JA, Ancian P. Newman NJ, et al. Among authors: sahel ja. BMC Neurol. 2022 Jul 12;22(1):257. doi: 10.1186/s12883-022-02787-y. BMC Neurol. 2022. PMID: 35820885 Free PMC article.
Lenadogene nolparvovec was administered by intravitreal injection (IVT) in LHON patients harboring the m.11778G > A ND4 mutation in a clinical development program including one phase 1/2 study (REVEAL), three phase 3 pivotal studies (REVERSE, RESCUE, REFLECT), and one long- …
Lenadogene nolparvovec was administered by intravitreal injection (IVT) in LHON patients harboring the m.11778G > A ND4 mutation in a cli …
Selective neural coding of object, feature, and geometry spatial cues in humans.
Ramanoël S, Durteste M, Bizeul A, Ozier-Lafontaine A, Bécu M, Sahel JA, Habas C, Arleo A. Ramanoël S, et al. Among authors: sahel ja. Hum Brain Mapp. 2022 Dec 1;43(17):5281-5295. doi: 10.1002/hbm.26002. Epub 2022 Jul 1. Hum Brain Mapp. 2022. PMID: 35776524 Free PMC article.
However, this dual-system hypothesis is based on paradigms that presented spatial cues conveying either conflicting or ambiguous spatial information and that used the term landmark to refer to both discrete three-dimensional objects and wall features. ...
However, this dual-system hypothesis is based on paradigms that presented spatial cues conveying either conflicting or ambiguous spatial inf …
Human corneal stromal stem cells express anti-fibrotic microRNA-29a and 381-5p - A robust cell selection tool for stem cell therapy of corneal scarring.
Yam GH, Yang T, Geary ML, Santra M, Funderburgh M, Rubin E, Du Y, Sahel JA, Jhanji V, Funderburgh JL. Yam GH, et al. Among authors: sahel ja. J Adv Res. 2023 Mar;45:141-155. doi: 10.1016/j.jare.2022.05.008. Epub 2022 May 25. J Adv Res. 2023. PMID: 35623612 Free PMC article.

MicroRNAs expressed > 20 folds in "healing" EVs (P < 0.05) were subject to enriched gene ontology (GO) term analysis. MiRNA groups with predictive regulation on inflammatory and fibrotic signalling were studied by mimic transfection to (1) mouse macrophages (RAW264.7

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MicroRNAs expressed > 20 folds in "healing" EVs (P < 0.05) were subject to enriched gene ontology (GO) term analysis. MiRNA gro

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New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies.
Pulman J, Sahel JA, Dalkara D. Pulman J, et al. Among authors: sahel ja. CRISPR J. 2022 Jun;5(3):377-388. doi: 10.1089/crispr.2021.0141. Epub 2022 May 3. CRISPR J. 2022. PMID: 35506982 Free PMC article. Review.
Gene therapy (GT) has emerged as an exciting treatment modality with the potential to provide long-term benefit to patients. Today, gene addition is the most straightforward GT for autosomal recessive IRDs. ...
Gene therapy (GT) has emerged as an exciting treatment modality with the potential to provide long-term benefit to patients. Today, g …
In vivo optogenetic stimulation of the primate retina activates the visual cortex after long-term transduction.
Chaffiol A, Provansal M, Joffrois C, Blaize K, Labernede G, Goulet R, Burban E, Brazhnikova E, Duebel J, Pouget P, Sahel JA, Picaud S, Arcizet F, Gauvain G. Chaffiol A, et al. Among authors: sahel ja. Mol Ther Methods Clin Dev. 2021 Nov 22;24:1-10. doi: 10.1016/j.omtm.2021.11.009. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2021. PMID: 34977267 Free PMC article.
But applications to human patients suffering from retinal diseases leading to blindness raise important concerns on the long-term functional expression of optogenes and the efficient signal transmission to higher visual centers. ...Using synaptic blockers, we isolated the …
But applications to human patients suffering from retinal diseases leading to blindness raise important concerns on the long-term fun …
33 results