Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes

por McClements, Michelle E., MacLaren, Robert E.
Publicado 2017

A Review of CRISPR Tools for Treating Usher Syndrome: Applicability, Safety, Efficiency, and In Vivo Delivery

por Major, Lauren, McClements, Michelle E., MacLaren, Robert E.
Publicado 2023

CRISPR-Cas9 DNA Base-Editing and Prime-Editing

por Kantor, Ariel, McClements, Michelle E., MacLaren, Robert E.
Publicado 2020

Therapy Approaches for Stargardt Disease

por Piotter, Elena, McClements, Michelle E, MacLaren, Robert E
Publicado 2021

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina

por Salman, Ahmed, McClements, Michelle E., MacLaren, Robert E.
Publicado 2021

The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems—A Systematic Review

por Piotter, Elena, McClements, Michelle E., MacLaren, Robert E.
Publicado 2022

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin

por Kaukonen, Maria, McClements, Michelle E., MacLaren, Robert E.
Publicado 2022

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome

por Major, Lauren, McClements, Michelle E., MacLaren, Robert E.
Publicado 2022

Minicircle Delivery to the Neural Retina as a Gene Therapy Approach

por Staurenghi, Federica, McClements, Michelle E., Salman, Ahmed, MacLaren, Robert E.
Publicado 2022

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances

por McClements, Michelle E., Staurenghi, Federica, MacLaren, Robert E., Cehajic-Kapetanovic, Jasmina
Publicado 2020

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases

por Hansen, Silja, McClements, Michelle E., Corydon, Thomas J., MacLaren, Robert E.
Publicado 2023

Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?

por Bellingrath, Julia-Sophia, McClements, Michelle E., Fischer, M. Dominik, MacLaren, Robert E.
Publicado 2023

Assessment of AAV Dual Vector Safety in the Abca4(−)(/)(−) Mouse Model of Stargardt Disease

por McClements, Michelle E., Barnard, Alun R., Charbel Issa, Peter, MacLaren, Robert E.
Publicado 2020

CRISPR Interference–Potential Application in Retinal Disease

por Peddle, Caroline F., Fry, Lewis E., McClements, Michelle E., MacLaren, Robert E.
Publicado 2020

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

por Martinez-Fernandez de la Camara, Cristina, McClements, Michelle E., MacLaren, Robert E.
Publicado 2021

An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy

por McClements, Michelle E., Butt, Anum, Piotter, Elena, Peddle, Caroline F., MacLaren, Robert E.
Publicado 2021

In Silico Analysis of Pathogenic CRB1 Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention

por Bellingrath, Julia-Sophia, McClements, Michelle E., Kaukonen, Maria, Fischer, Manuel Dominik, MacLaren, Robert E.
Publicado 2021

Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations

por Salman, Ahmed, Kantor, Ariel, McClements, Michelle E., Marfany, Gemma, Trigueros, Sonia, MacLaren, Robert E.
Publicado 2022

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina

por Patrício, Maria I., Barnard, Alun R., Orlans, Harry O., McClements, Michelle E., MacLaren, Robert E.
Publicado 2017

Molecular Strategies for RPGR Gene Therapy

por Cehajic Kapetanovic, Jasmina, McClements, Michelle E, Martinez-Fernandez de la Camara, Cristina, MacLaren, Robert E
Publicado 2019