Cargando…

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

INTRODUCTION: Choroideremia is an X-linked inherited retinal degeneration resulting from mutations in the CHM gene, encoding Rab escort protein-1 (REP1), a protein regulating intracellular vesicular transport. Loss-of-function mutations in CHM lead to progressive loss of retinal pigment epithelium (...

Descripción completa

Detalles Bibliográficos
Autores principales:	Han, Ruofan Connie, Fry, Lewis E., Kantor, Ariel, McClements, Michelle E., Xue, Kanmin, MacLaren, Robert E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7610829/ https://www.ncbi.nlm.nih.gov/pubmed/34040899 http://dx.doi.org/10.1080/21678707.2021.1882300

Ejemplares similares

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
por: Chandler, Laurel C., et al.
Publicado: (2021)

Expression of Rab Prenylation Pathway Genes and Relation to Disease Progression in Choroideremia
por: Fry, Lewis E., et al.
Publicado: (2021)

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial
por: MacLaren, Robert E., et al.
Publicado: (2023)

Characterizing the Natural History of Visual Function in Choroideremia Using Microperimetry and Multimodal Retinal Imaging
por: Jolly, Jasleen K., et al.
Publicado: (2017)

 Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes
por: McClements, Michelle E., et al.
Publicado: (2017)

Genome-Editing Strategies for Treating Human Retinal Degenerations
por: Quinn, Joel, et al.
Publicado: (2021)

The Spectrum of CHM Gene Mutations in Choroideremia and Their Relationship to Clinical Phenotype
por: Simunovic, Matthew P., et al.
Publicado: (2016)

Molecular Therapies for Choroideremia
por: Cehajic Kapetanovic, Jasmina, et al.
Publicado: (2019)

Recent advances and future prospects in choroideremia
por: Zinkernagel, Martin S, et al.
Publicado: (2015)

CRISPR-Cas9 DNA Base-Editing and Prime-Editing
por: Kantor, Ariel, et al.
Publicado: (2020)

Correlation of Optical Coherence Tomography and Autofluorescence in the Outer Retina and Choroid of Patients With Choroideremia
por: Xue, Kanmin, et al.
Publicado: (2016)

The Biological Activity of AAV Vectors for Choroideremia Gene Therapy Can Be Measured by In Vitro Prenylation of RAB6A
por: Patrício, Maria I., et al.
Publicado: (2018)

Accurate Quantification of AAV Vector Genomes by Quantitative PCR
por: Martinez-Fernandez de la Camara, Cristina, et al.
Publicado: (2021)

Choroideremia: The Endpoint Endgame
por: Abdalla Elsayed, Maram E. A., et al.
Publicado: (2023)

Assessment of AAV Dual Vector Safety in the Abca4(−)(/)(−) Mouse Model of Stargardt Disease
por: McClements, Michelle E., et al.
Publicado: (2020)

Outcome of Full-Thickness Macular Hole Surgery in Choroideremia
por: Talib, Mays, et al.
Publicado: (2017)

Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations
por: Salman, Ahmed, et al.
Publicado: (2022)

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina
por: Patrício, Maria I., et al.
Publicado: (2017)

Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids
por: McClements, Michelle E., et al.
Publicado: (2022)

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration
por: McClements, Michelle E., et al.
Publicado: (2021)

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo
por: Tolmachova, Tanya, et al.
Publicado: (2013)

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?
por: Bellingrath, Julia-Sophia, et al.
Publicado: (2023)

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy
por: Chandler, Laurel C., et al.
Publicado: (2020)

A Qualitative and Quantitative Assessment of Fundus Autofluorescence Patterns in Patients With Choroideremia
por: Jolly, Jasleen K., et al.
Publicado: (2016)

Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells
por: Dreismann, Anna K., et al.
Publicado: (2021)

CRISPR Interference–Potential Application in Retinal Disease
por: Peddle, Caroline F., et al.
Publicado: (2020)

AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
por: Vasireddy, Vidyullatha, et al.
Publicado: (2013)

Structural and Functional Characteristics of Color Vision Changes in Choroideremia
por: Jolly, Jasleen K., et al.
Publicado: (2021)

Low Luminance Visual Acuity and Low Luminance Deficit in Choroideremia and RPGR-Associated Retinitis Pigmentosa
por: Wood, Laura J., et al.
Publicado: (2021)

Longitudinal Study to Assess the Quantitative Use of Fundus Autofluorescence for Monitoring Disease Progression in Choroideremia
por: Dubis, Adam M, et al.
Publicado: (2021)

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina
por: Hickey, D G, et al.
Publicado: (2017)

Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
por: Vasireddy, Vidyullatha, et al.
Publicado: (2015)

Correlation Between Fundus Autofluorescence Pattern and Retinal Function on Microperimetry in Choroideremia
por: Poli, Federica E., et al.
Publicado: (2023)

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina
por: Salman, Ahmed, et al.
Publicado: (2021)

Therapy Approaches for Stargardt Disease
por: Piotter, Elena, et al.
Publicado: (2021)

The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems—A Systematic Review
por: Piotter, Elena, et al.
Publicado: (2022)

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin
por: Kaukonen, Maria, et al.
Publicado: (2022)

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome
por: Major, Lauren, et al.
Publicado: (2022)

A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery
por: Major, Lauren, et al.
Publicado: (2023)

RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences
por: Fry, Lewis E., et al.
Publicado: (2020)

Cannot write session to /tmp/vufind_sessions/sess_maofvuif5d6jk7q1pilt7dqf2p