Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina

Although adeno-associated viral (AAV) vector-mediated retinal gene therapies have demonstrated efficacy, the mechanisms underlying dose-dependent retinal inflammation remain poorly understood. Here we present a quantitative analysis of cellular immune response to subretinal AAV gene therapy in mice...

Bibliografski detalji
Glavni autori:	Chandler, LC, McClements, ME, Yusuf, IH, Martinez-Fernandez de la Camara, C, MacLaren, RE, Xue, K
Format:	Journal article
Jezik:	English
Izdano:	Cell Press 2021

Slični predmeti

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
od: Laurel C. Chandler, i dr.
Izdano: (2021-09-01)

Cell-mediated inflammatory response to AAV gene therapy in the retina
od: Chandler, LC, i dr.
Izdano: (2020)

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
od: Han, RC, i dr.
Izdano: (2021)

Insights on the regeneration potential of Müller glia in the mammalian retina
od: Salman, A, i dr.
Izdano: (2021)

Minicircle delivery to the neural retina as a gene therapy approach
od: Staurenghi, F, i dr.
Izdano: (2022)

Inclusion of the woodchuck hepatitis virus posttranscriptional regulatory element enhances AAV2-driven transduction of mouse and human retina
od: Patrício, M, i dr.
Izdano: (2017)

Adeno-associated virus (AAV) dual vector strategies for gene therapy encoding large transgenes
od: McClements, M, i dr.
Izdano: (2017)

Promoter orientation within an AAV-CRISPR vector affects Cas9 expression and gene editing efficiency
od: Fry, LE, i dr.
Izdano: (2020)

Non-viral delivery of CRIPSR/Cas cargo to the retina using nanoparticles: current possibilities, challenges, and limitations
od: Salman, A, i dr.
Izdano: (2022)

Inclusion of the Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element Enhances AAV2-Driven Transduction of Mouse and Human Retina
od: Maria I. Patrício, i dr.
Izdano: (2017-03-01)

Tropism of engineered and evolved recombinant AAV serotypes in the rd1 mouse and ex vivo primate retina
od: Hickey, DG, i dr.
Izdano: (2017)

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina
od: Ahmed Salman, i dr.
Izdano: (2021-07-01)

Enhancement of adeno-associated viral (AAV) gene therapy using hydroxychloroquine in murine and human tissues
od: Chandler, L, i dr.
Izdano: (2019)

Optogenetic Gene Therapy for the Degenerate Retina: Recent Advances
od: Michelle E. McClements, i dr.
Izdano: (2020-11-01)

Codon-optimized RPGR improves stability and efficacy of AAV8 gene therapy in two mouse models of X-linked retinitis pigmentosa
od: Fischer, MD, i dr.
Izdano: (2017)

Tetradecanoylphorbol-13-acetate (TPA) significantly increases AAV2/5 transduction of human neuronal cells in vitro
od: You, Q, i dr.
Izdano: (2012)

Gene Therapies in Clinical Development to Treat Retinal Disorders
od: McClements, ME, i dr.
Izdano: (2024)

RNA editing for Usher syndrome using CRISPR-Cas13
od: Fry, LE, i dr.
Izdano: (2020)

Long-term restoration of visual function in end-stage retinal degeneration using subretinal human melanopsin gene therapy
od: De Silva, SR, i dr.
Izdano: (2017)

An AAV dual vector strategy ameliorates the Stargardt phenotype in adult Abca4−/− mice
od: McClements, ME, i dr.
Izdano: (2018)

Tropisms of AAV for subretinal delivery to the neonatal mouse retina and its application for in vivo rescue of developmental photoreceptor disorders.
od: Satoshi Watanabe, i dr.
Izdano: (2013-01-01)

Technique of retinal gene therapy: delivery of viral vector into the subretinal space
od: Xue, K, i dr.
Izdano: (2017)

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
od: Orlans, HO, i dr.
Izdano: (2021)

AAV2/8 Anti-angiogenic Gene Therapy Using Single-Chain Antibodies Inhibits Murine Choroidal Neovascularization
od: Chris P. Hughes, i dr.
Izdano: (2019-06-01)

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

RNA editing as a therapeutic approach for retinal gene therapy requiring long coding sequences
od: Fry, LE, i dr.
Izdano: (2020)

Immunomodulatory effects of hydroxychloroquine and chloroquine in viral infections and their potential application in retinal gene therapy
od: Chandler, L, i dr.
Izdano: (2020)

Gene therapy for retinal disease.
od: McClements, M, i dr.
Izdano: (2013)

Non-Viral Delivery of CRISPR/Cas Cargo to the Retina Using Nanoparticles: Current Possibilities, Challenges, and Limitations
od: Ahmed Salman, i dr.
Izdano: (2022-09-01)

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial
od: MacLaren, RE, i dr.
Izdano: (2023)

Choroideremia: molecular mechanisms and development of AAV gene therapy
od: Patrício, M, i dr.
Izdano: (2018)

Envisioning the development of a CRISPR-Cas mediated base editing strategy for a patient with a novel pathogenic CRB1 single nucleotide variant
od: Bellingrath, J-S, i dr.
Izdano: (2022)

The influence of subretinal injection pressure on the microstructure of the monkey retina.
od: Kosuke Takahashi, i dr.
Izdano: (2018-01-01)

CDHR1-related late-onset macular dystrophy: further insights
od: Yusuf, IH, i dr.
Izdano: (2020)

Association of a novel intronic variant in RPGR with hypomorphic phenotype of x-linked retinitis pigmentosa
od: Cehajic-Kapetanovic, J, i dr.
Izdano: (2020)

Genome-editing strategies for treating human retinal degenerations
od: Quinn, J, i dr.
Izdano: (2021)

Functional expression of Rab escort protein 1 following AAV2-mediated gene delivery in the retina of choroideremia mice and human cells ex vivo
od: Tolmachova, T, i dr.
Izdano: (2013)

Storage of Human Fetal Retina in Optisol Prior to Subretinal Transplantation
od: David A. Diloreto, i dr.
Izdano: (1996-09-01)

Selective retina therapy for subretinal fluid associated with choroidal nevus
od: Manabu Yamamoto, i dr.
Izdano: (2020-09-01)

Tetradecanoylphorbol-13-acetate (TPA) significantly increases AAV2/5 transduction of human neuronal cells in vitro.
od: You, Q, i dr.
Izdano: (2012)