Gene Therapies in Clinical Development to Treat Retinal Disorders

Gene Therapies in Clinical Development to Treat Retinal Disorders

Gene therapies have emerged as promising treatments in clinical development for various retinal disorders, offering hope to patients with inherited degenerative eye conditions. Several gene therapies have already shown remarkable success in clinical trials, with significant improvements observed in...

Full description

Bibliographic Details
Main Authors:	McClements, ME, Elsayed, MEAA, Major, L, de la Camara, CM, MacLaren, RE
Format:	Journal article
Language:	English
Published:	Springer 2024

Similar Items

Gene therapy for retinal disease.
by: McClements, M, et al.
Published: (2013)

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

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

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

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

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

Gene therapy for retinal dystrophies
by: Issa, P, et al.
Published: (2012)

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

Immunomodulatory effects of hydroxychloroquine and chloroquine in viral infections and their potential application in retinal gene therapy
by: Chandler, L, et al.
Published: (2020)

Adeno-associated virus (AAV) dual vector strategies for gene therapy encoding large transgenes
by: McClements, M, et al.
Published: (2017)

Cell-mediated inflammatory response to AAV gene therapy in the retina
by: Chandler, LC, et al.
Published: (2020)

Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration
by: Fry, LE, et al.
Published: (2025)

Clinical applications of retinal gene therapy.
by: Lipinski, D, et al.
Published: (2013)

An analysis of retinal gene therapy clinical trials.
by: Maclaren, R
Published: (2009)

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

Gene therapy for age-related macular degeneration
by: MacLaren, RE
Published: (2015)

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

RPGR gene therapy presents challenges in cloning the coding sequence
by: Martinez-Fernandez De La Camara, C, et al.
Published: (2019)

Insights on the regeneration potential of Müller glia in the mammalian retina
by: Salman, A, et al.
Published: (2021)

Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy
by: De Silva, SR, et al.
Published: (2017)

Technique of retinal gene therapy: delivery of viral vector into the subretinal space
by: Xue, K, et al.
Published: (2017)

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

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

Structural and functional recovery following limited iatrogenic macular detachment for retinal gene therapy.
by: Simunovic, M, et al.
Published: (2017)

[Gene therapy for retinal dystrophies].
by: Charbel Issa, P, et al.
Published: (2012)

RNA editing for Usher syndrome using CRISPR-Cas13
by: Fry, LE, et al.
Published: (2020)

Clinical characterization of retinitis pigmentosa associated with variants in SNRNP200
by: Yusuf, IH, et al.
Published: (2019)

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
by: Han, RC, et al.
Published: (2021)

Retinal gene therapy in patients with choroideremia: Initial fi ndings from a phase 1/2 clinical trial
by: MacLaren, R, et al.
Published: (2014)

Promoter orientation within an AAV-CRISPR vector affects Cas9 expression and gene editing efficiency
by: Fry, LE, et al.
Published: (2020)

Single-cell transcriptomic analysis of retinal immune regulation and blood-retinal barrier function during experimental autoimmune uveitis
by: Quinn, J, et al.
Published: (2024)

"Genetic and clinical findings in an ethnically diverse retinitis pigmentosa cohort associated with pathogenic variants in EYS"
by: Cundy, O, et al.
Published: (2020)

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

Visual acuity after retinal gene therapy for Choroideremia
by: Edwards, T, et al.
Published: (2016)

In silico analysis of pathogenic CRB1 single nucleotide variants and their amenability to base editing as a potential lead for therapeutic intervention
by: Bellingrath, J-S, et al.
Published: (2021)

Non-viral retinal gene therapy: A review
by: Issa, P, et al.
Published: (2012)

Hypotrichosis and juvenile macular dystrophy caused by CDH3 mutation: A candidate disease for retinal gene therapy
by: Singh, M, et al.
Published: (2016)

Non-viral retinal gene therapy: a review.
by: Charbel Issa, P, et al.
Published: (2012)

Neuroprotective gene therapy for the treatment of inherited retinal degeneration.
by: Buch, P, et al.
Published: (2007)

Non-viral delivery of CRIPSR/Cas cargo to the retina using nanoparticles: current possibilities, challenges, and limitations
by: Salman, A, et al.
Published: (2022)