RPGR gene therapy presents challenges in cloning the coding sequence

RPGR gene therapy presents challenges in cloning the coding sequence

<p><strong>Introduction:</strong> Currently, there are three Phase I/II clinical trials based on gene therapy ongoing to test different AAV.RPGR or deleted RPGR vectors on patients affected by X-linked retinitis pigmentosa. These three vectors differ in the adeno-associated viral (...

Descrizione completa

Dettagli Bibliografici
Autori principali:	Martinez-Fernandez De La Camara, C, Cehajic-Kapetanovic, J, MacLaren, RE
Natura:	Journal article
Lingua:	English
Pubblicazione:	Taylor and Francis 2019

Documenti analoghi

Clinical applications of microperimetry in RPGR-related retinitis pigmentosa: a review
di: Buckley, TMW, et al.
Pubblicazione: (2021)

Characterizing visual fields in RPGR related retinitis pigmentosa using octopus static-automated perimetry
di: Buckley, TMW, et al.
Pubblicazione: (2022)

Association of a novel intronic variant in RPGR with hypomorphic phenotype of x-linked retinitis pigmentosa
di: Cehajic-Kapetanovic, J, et al.
Pubblicazione: (2020)

Microperimetry as an outcome measure in RPGR-associated retinitis pigmentosa clinical trials
di: Taylor, LJ, et al.
Pubblicazione: (2023)

Low luminance visual acuity and low luminance deficit in choroideremia and RPGR-associated retinitis pigmentosa
di: Wood, LJ, et al.
Pubblicazione: (2021)

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
di: Cehajic-Kapetanovic, J, et al.
Pubblicazione: (2020)

Test‐retest repeatability of microperimetry in patients with retinitis pigmentosa caused by mutations in RPGR
di: Buckley, TMW, et al.
Pubblicazione: (2020)

Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa
di: Fischer, MD, et al.
Pubblicazione: (2017)

Robotising vitreoretinal surgeries
di: Mi, H, et al.
Pubblicazione: (2024)

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances
di: Michelle E. McClements, et al.
Pubblicazione: (2020-11-01)

Current and Future Landscape in Genetic Therapies for Leber Hereditary Optic Neuropathy
di: Hoda Shamsnajafabadi, et al.
Pubblicazione: (2023-08-01)

A novel splice-site variant in CDH23 in a patient with Usher syndrome type 1
di: Menghini, M, et al.
Pubblicazione: (2019)

High symmetry of visual acuity and visual fields in RPGR-linked retinitis pigmentosa
di: Bellingrath, J-S, et al.
Pubblicazione: (2017)

Changes in retinal sensitivity associated with cotoretigene toliparvovec in X-linked retinitis pigmentosa with RPGR gene variations
di: von Krusenstiern, L, et al.
Pubblicazione: (2023)

Microperimetry hill of vision and volumetric measures of retinal sensitivity
di: Josan, AS, et al.
Pubblicazione: (2021)

Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy
di: Borchert, GA, et al.
Pubblicazione: (2024)

Inner retinal thickening affects microperimetry thresholds in the presence of photoreceptor thinning in patients with RPGR retinitis pigmentosa
di: Jolly, JK, et al.
Pubblicazione: (2020)

RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences
di: Fry, LE, et al.
Pubblicazione: (2020)

Gene therapy for age-related macular degeneration
di: MacLaren, RE
Pubblicazione: (2015)

Genome-editing strategies for treating human retinal degenerations
di: Quinn, J, et al.
Pubblicazione: (2021)

The Role of RPGR and Its Interacting Proteins in Ciliopathies
di: Sarita Rani Patnaik, et al.
Pubblicazione: (2015-01-01)

Compound dominant-null heterozygosity in a family with RP1-related retinal dystrophy
di: Thomas M.W. Buckley, et al.
Pubblicazione: (2022-12-01)

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
di: Orlans, HO, et al.
Pubblicazione: (2021)

Age-related macular degeneration: suitability of optogenetic therapy for geographic atrophy
di: Grace A. Borchert, et al.
Pubblicazione: (2024-07-01)

Gene therapy for choroideremia using an adeno-associated viral vector encoding Rab escort protein 1: the REGENERATE open-label trial
di: Jasmina Cehajic-Kapetanovic, et al.
Pubblicazione: (2024-05-01)

Potential CRISPR base editing therapeutic options in a Sorsby fundus dystrophy patient
di: Elsayed, MEAA, et al.
Pubblicazione: (2022)

Gene Therapies in Clinical Development to Treat Retinal Disorders
di: McClements, ME, et al.
Pubblicazione: (2024)

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
di: Chandler, LC, et al.
Pubblicazione: (2021)

X-linked dominant RPGR gene mutation in a familial Coats angiomatosis
di: Marcella Nebbioso, et al.
Pubblicazione: (2021-01-01)

Real-world six-month outcomes in patients switched to faricimab following partial response to anti-VEGF therapy for neovascular age-related macular degeneration and diabetic macular oedema
di: Borchert, GA, et al.
Pubblicazione: (2024)

Visual Acuity by Decade in 139 Males with RPGR-Associated Retinitis Pigmentosa
di: Samantha R. De Silva, DPhil, FRCOphth, et al.
Pubblicazione: (2024-03-01)

Identification of circular RNAs hosted by the RPGR ORF15 genomic locus
di: Tatyana Appelbaum, et al.
Pubblicazione: (2023-12-01)

Novel mutations of RPGR in Chinese families with X-linked retinitis pigmentosa
di: Zhimeng Zhang, et al.
Pubblicazione: (2019-11-01)

Novel mutations of RPGR in Chinese retinitis pigmentosa patients and the genotype-phenotype correlation.
di: Liping Yang, et al.
Pubblicazione: (2014-01-01)

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases
di: Xue, K, et al.
Pubblicazione: (2020)

Recent advances and future prospects in choroideremia
di: Zinkernagel MS, et al.
Pubblicazione: (2015-11-01)

Cloning of Deleted Sequences (CODE): A Genomic Subtraction Method for Enriching and Cloning Deleted Sequences
di: J. Li, et al.
Pubblicazione: (2001-10-01)

Minicircle delivery to the neural retina as a gene therapy approach
di: Staurenghi, F, et al.
Pubblicazione: (2022)

A novel mutation of RPGR in a Chinese family with X-linked retinitis pigmentosa
di: Hui-Hui Sun, et al.
Pubblicazione: (2022-09-01)

Structural and functional plasticity of subcellular tethering, targeting and processing of RPGRIP1 by RPGR isoforms
di: Hemangi Patil, et al.
Pubblicazione: (2012-02-01)