Gene therapy for retinal disease

von McClements, Michelle E, MacLaren, Robert E
Veröffentlicht 2013


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

von McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Yale J Biol Med (2017)

CRISPR-Cas9 DNA Base-Editing and Prime-Editing

von Kantor, Ariel, McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Int J Mol Sci (2020)

Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration

von Fry, Lewis E., McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in JAMA Ophthalmol (2021)

A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts

von McClements, Michelle E., Charbel Issa, Peter, Blouin, Véronique, MacLaren, Robert E.
Veröffentlicht in J Genet Syndr Gene Ther (2016)

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances

von McClements, Michelle E., Staurenghi, Federica, MacLaren, Robert E., Cehajic-Kapetanovic, Jasmina
Veröffentlicht in Front Neurosci (2020)

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

von McClements, Michelle E., Barnard, Alun R., Charbel Issa, Peter, MacLaren, Robert E.
Veröffentlicht in Transl Vis Sci Technol (2020)

CRISPR Interference–Potential Application in Retinal Disease

von Peddle, Caroline F., Fry, Lewis E., McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Int J Mol Sci (2020)

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

von Martinez-Fernandez de la Camara, Cristina, McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Genes (Basel) (2021)

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

von McClements, Michelle E., Butt, Anum, Piotter, Elena, Peddle, Caroline F., MacLaren, Robert E.
Veröffentlicht in Mol Vis (2021)

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

von Patrício, Maria I., Barnard, Alun R., Orlans, Harry O., McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Mol Ther Nucleic Acids (2017)

Molecular Strategies for RPGR Gene Therapy

von Cehajic Kapetanovic, Jasmina, McClements, Michelle E, Martinez-Fernandez de la Camara, Cristina, MacLaren, Robert E
Veröffentlicht in Genes (Basel) (2019)

RNA Editing as a Therapeutic Approach for Retinal Gene Therapy Requiring Long Coding Sequences

von Fry, Lewis E., Peddle, Caroline F., Barnard, Alun R., McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in Int J Mol Sci (2020)

Association of a Novel Intronic Variant in RPGR With Hypomorphic Phenotype of X-Linked Retinitis Pigmentosa

von Cehajic-Kapetanovic, Jasmina, McClements, Michelle E., Whitfield, Jennifer, Shanks, Morag, Clouston, Penny, MacLaren, Robert E.
Veröffentlicht in JAMA Ophthalmol (2020)

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

von Han, Ruofan Connie, Fry, Lewis E., Kantor, Ariel, McClements, Michelle E., Xue, Kanmin, MacLaren, Robert E.
Veröffentlicht in Expert Opin Orphan Drugs (2021)

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration

von McClements, Michelle E., Staurenghi, Federica, Visel, Meike, Flannery, John G., MacLaren, Robert E., Cehajic-Kapetanovic, Jasmina
Veröffentlicht in Biomed Res Int (2021)

Immunomodulatory Effects of Hydroxychloroquine and Chloroquine in Viral Infections and Their Potential Application in Retinal Gene Therapy

von Chandler, Laurel C., Yusuf, Imran H., McClements, Michelle E., Barnard, Alun R., MacLaren, Robert E., Xue, Kanmin
Veröffentlicht in Int J Mol Sci (2020)

Promoter Orientation within an AAV-CRISPR Vector Affects Cas9 Expression and Gene Editing Efficiency

von Fry, Lewis E., Peddle, Caroline F., Stevanovic, Marta, Barnard, Alun R., McClements, Michelle E., MacLaren, Robert E.
Veröffentlicht in CRISPR J (2020)

Analysis of Early Cone Dysfunction in an In Vivo Model of Rod-Cone Dystrophy

von Hassall, Mark M., McClements, Michelle E., Barnard, Alun R., Patrício, Maria I., Aslam, Sher A., Maclaren, Robert E.
Veröffentlicht in Int J Mol Sci (2020)

Genome-Editing Strategies for Treating Human Retinal Degenerations

von Quinn, Joel, Musa, Ayesha, Kantor, Ariel, McClements, Michelle E., Cehajic-Kapetanovic, Jasmina, MacLaren, Robert E., Xue, Kanmin
Veröffentlicht in Hum Gene Ther (2021)