Promoter orientation within an AAV-CRISPR vector affects Cas9 expression and gene editing efficiency

Promoter orientation within an AAV-CRISPR vector affects Cas9 expression and gene editing efficiency

Adeno-associated virus (AAV) vectors have been widely adopted for delivery of CRISPR-Cas components, especially for therapeutic gene editing. For a single vector system, both the Cas9 and guide RNA (gRNA) are encoded within a single transgene, usually from separate promoters. Careful design of this...

書誌詳細
主要な著者:	Fry, LE, Peddle, CF, Stevanovic, M, Barnard, AR, McClements, ME, MacLaren, RE
フォーマット:	Journal article
言語:	English
出版事項:	Mary Ann Liebert Inc 2020

類似資料

RNA editing for Usher syndrome using CRISPR-Cas13
著者:: Fry, LE, 等
出版事項: (2020)

RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences
著者:: Fry, LE, 等
出版事項: (2020)

Adeno-associated virus (AAV) dual vector strategies for gene therapy encoding large transgenes
著者:: McClements, M, 等
出版事項: (2017)

Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration
著者:: Fry, LE, 等
出版事項: (2025)

Envisioning the development of a CRISPR-Cas mediated base editing strategy for a patient with a novel pathogenic CRB1 single nucleotide variant
著者:: Bellingrath, J-S, 等
出版事項: (2022)

An AAV dual vector strategy ameliorates the Stargardt phenotype in adult Abca4−/− mice
著者:: McClements, ME, 等
出版事項: (2018)

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
著者:: Han, RC, 等
出版事項: (2021)

Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa
著者:: Peddle, CF
出版事項: (2021)

Cell-mediated inflammatory response to AAV gene therapy in the retina
著者:: Chandler, LC, 等
出版事項: (2020)

Inclusion of the woodchuck hepatitis virus posttranscriptional regulatory element enhances AAV2-driven transduction of mouse and human retina
著者:: Patrício, M, 等
出版事項: (2017)

Comparison of CRISPR-Cas13b RNA base editing approaches for USH2A-associated inherited retinal degeneration
著者:: Lewis E. Fry, 等
出版事項: (2025-02-01)

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
著者:: Chandler, LC, 等
出版事項: (2021)

Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa
著者:: Fischer, MD, 等
出版事項: (2017)

In silico analysis of pathogenic CRB1 single nucleotide variants and their amenability to base editing as a potential lead for therapeutic intervention
著者:: Bellingrath, J-S, 等
出版事項: (2021)

Non-viral delivery of CRIPSR/Cas cargo to the retina using nanoparticles: current possibilities, challenges, and limitations
著者:: Salman, A, 等
出版事項: (2022)

Insights on the regeneration potential of Müller glia in the mammalian retina
著者:: Salman, A, 等
出版事項: (2021)

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria
著者:: Daelyn Y. Richards, 等
出版事項: (2020-06-01)

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina
著者:: Hickey, DG, 等
出版事項: (2017)

Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations
著者:: Ahmed Salman, 等
出版事項: (2022-09-01)

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina
著者:: Maria I. Patrício, 等
出版事項: (2017-03-01)

Tetradecanoylphorbol-13-acetate (TPA) significantly increases AAV2/5 transduction of human neuronal cells in vitro
著者:: You, Q, 等
出版事項: (2012)

Genome-editing strategies for treating human retinal degenerations
著者:: Quinn, J, 等
出版事項: (2021)

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
著者:: Orlans, HO, 等
出版事項: (2021)

The biological activity of AAV vectors for choroideremia gene therapy can be measured by in vitro prenylation of RAB6A
著者:: Patrício, M, 等
出版事項: (2018)

Minicircle delivery to the neural retina as a gene therapy approach
著者:: Staurenghi, F, 等
出版事項: (2022)

Choroideremia: molecular mechanisms and development of AAV gene therapy
著者:: Patrício, M, 等
出版事項: (2018)

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
著者:: Wang, D, 等
出版事項: (2021)

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
著者:: Wang, Dan, 等
出版事項: (2022)

Potential CRISPR base editing therapeutic options in a Sorsby fundus dystrophy patient
著者:: Elsayed, MEAA, 等
出版事項: (2022)

Gene Therapy for Choroideremia Using an Adeno-Associated Viral (AAV) Vector.
著者:: Barnard, A, 等
出版事項: (2014)

Genome Editing With TALEN, CRISPR-Cas9 and CRISPR-Cas12a in Combination With AAV6 Homology Donor Restores T Cell Function for XLP
著者:: Benjamin C. Houghton, 等
出版事項: (2022-05-01)

Impact of vital dyes on cell viability and transduction efficiency of AAV vectors used in retinal gene therapy surgery: an in vitro and in vivo analysis
著者:: Salvetti, A, 等
出版事項: (2017)

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
著者:: Giridhar Murlidharan, 等
出版事項: (2016-01-01)

Future Perspectives of Prime Editing for the Treatment of Inherited Retinal Diseases
著者:: Silja Hansen, 等
出版事項: (2023-01-01)

Gene therapy for retinal disease.
著者:: McClements, M, 等
出版事項: (2013)

Optimizing an AAV-CRISPR system for retinal gene therapy
著者:: Stevanovic, M
出版事項: (2019)

AAV2/2-CBA-REP1 Vector with WPRE Provides Functional Expression of the Transgene in Choroideremia Mouse Knock-Out and Patient Cells
著者:: Tolmachova, T, 等
出版事項: (2012)

CRISPR/Cas9 gene editing in the West Nile Virus vector, Culex quinquefasciatus Say
著者:: Anderson, ME, 等
出版事項: (2018)

Dynamic in vivo quantification of rod photoreceptor degeneration using fluorescent reporter mouse models of retinitis pigmentosa
著者:: Orlans, HO, 等
出版事項: (2019)

A fragmented adeno-associated viral dual vector strategy for treatment of diseases caused by mutations in large genes leads to expression of hybrid transcripts
著者:: McClements, M, 等
出版事項: (2016)