Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa

Development of all-in-one CRISPR/Cas9 and CRISPRi AAV constructs to treat autosomal dominant retinitis pigmentosa

<p>Dominant mutations in RHO (rhodopsin) are the most common cause of autosomal dominant retinitis pigmentosa (RHO-adRP). RHO-adRP causes progressive loss of rod cells, followed by cone cells, leading to blindness. This disease is a candidate for CRISPR gene editing, as reduction of mutant rho...

Cur síos iomlán

Sonraí bibleagrafaíochta
Príomhchruthaitheoir:	Peddle, CF
Rannpháirtithe:	MacLaren, RE
Formáid:	Tráchtas
Teanga:	English
Foilsithe / Cruthaithe:	2021
Ábhair:	Retinal degeneration Gene therapy CRISPR (Genetics) Retinitis pigmentosa

Míreanna comhchosúla

Rhodopsin mutations are scarcely implicated in autosomal recessive retinitis pigmentosa: A preliminary study of Egyptian retinitis pigmentosa patients
de réir: Reem Mebed, et al.
Foilsithe / Cruthaithe: (2015-10-01)

AAV-CRISPR/Cas9 Gene Editing Preserves Long-Term Vision in the P23H Rat Model of Autosomal Dominant Retinitis Pigmentosa
de réir: Saba Shahin, et al.
Foilsithe / Cruthaithe: (2022-04-01)

Herencia de la retinosis pigmentaria en la provincia Camagüey Inheritance of retinitis pigmentosa in the province of Camagüey
de réir: Elisa Dyce Gordon, et al.
Foilsithe / Cruthaithe: (1999-06-01)

Whole exome sequencing reveals novel EYS mutations in Chinese patients with autosomal recessive retinitis pigmentosa
de réir: Xiaoqiang Xiao, et al.
Foilsithe / Cruthaithe: (2019-01-01)

Genetic dissection of non-syndromic retinitis pigmentosa
de réir: Aarti Bhardwaj, et al.
Foilsithe / Cruthaithe: (2022-01-01)

Visual Acuity by Decade in 139 Males with RPGR-Associated Retinitis Pigmentosa
de réir: Samantha R. De Silva, DPhil, FRCOphth, et al.
Foilsithe / Cruthaithe: (2024-03-01)

Clinical and genetic features of Koreans with retinitis pigmentosa associated with mutations in rhodopsin
de réir: Young Hoon Jung, et al.
Foilsithe / Cruthaithe: (2023-08-01)

Stem cells set their sights on retinitis pigmentosa
de réir: Jeannette L Bennicelli, et al.
Foilsithe / Cruthaithe: (2013-08-01)

A patient with albinism and retinitis pigmentosa, a case report
de réir: Michalis Georgiou, et al.
Foilsithe / Cruthaithe: (2024-06-01)

Alteraciones psíquicas en niños con retinosis pigmentaria Psychical disorders in retinitis pigmentosa-affected children
de réir: Irene Quiñones Varela, et al.
Foilsithe / Cruthaithe: (1999-12-01)

Self-reported visual function and in-depth swept-source optical coherence tomography features of cystoid macular edema in retinitis pigmentosa
de réir: Celso Costa, et al.
Foilsithe / Cruthaithe: (2024-11-01)

Retinal Vascular Degeneration in the Transgenic P23H Rat Model of Retinitis Pigmentosa
de réir: Laura Fernández-Sánchez, et al.
Foilsithe / Cruthaithe: (2018-06-01)

Clinical Characteristics, Differential Diagnosis and Genetic Analysis of Concentric Retinitis Pigmentosa
de réir: Mei Nakahara, et al.
Foilsithe / Cruthaithe: (2021-03-01)

A novel mutation in PRPF31, causative of autosomal dominant retinitis pigmentosa, using the BGISEQ-500 sequencer
de réir: Yu Zheng, et al.
Foilsithe / Cruthaithe: (2018-01-01)

A novel homozygous TUB mutation associated with autosomal recessive retinitis pigmentosa in a consanguineous Chinese family
de réir: Wei Xu, et al.
Foilsithe / Cruthaithe: (2023-01-01)

Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development
de réir: Kevin Y. Wu, et al.
Foilsithe / Cruthaithe: (2023-02-01)

Myriocin Effect on Tvrm4 Retina, an Autosomal Dominant Pattern of Retinitis Pigmentosa
de réir: Ilaria Piano, et al.
Foilsithe / Cruthaithe: (2020-05-01)

Soluble CX3CL1-expressing retinal pigment epithelium cells protect rod photoreceptors in a mouse model of retinitis pigmentosa
de réir: Eric D. Jong, et al.
Foilsithe / Cruthaithe: (2023-08-01)

Retinitis Pigmentosa in Childhood: A Case Report
de réir: Erdal Eren, et al.
Foilsithe / Cruthaithe: (2008-06-01)

Thicknesses of the retina and choroid in children with retinitis pigmentosa
de réir: Cheng Li, et al.
Foilsithe / Cruthaithe: (2023-01-01)

Novel mutations in the 3-box motif of the BACK domain of KLHL7 associated with nonsyndromic autosomal dominant retinitis pigmentosa
de réir: Jin Kyun Oh, et al.
Foilsithe / Cruthaithe: (2019-12-01)

Trans-Sclera Electrical Stimulation Improves Retinal Function in a Mouse Model of Retinitis Pigmentosa
de réir: Feng Liu, et al.
Foilsithe / Cruthaithe: (2022-11-01)

Identification of two novel PRPF31 mutations in Chinese families with non‐syndromic autosomal dominant retinitis pigmentosa
de réir: Li Cao, et al.
Foilsithe / Cruthaithe: (2020-12-01)

Synthesis and Evaluation of Glucosyl-, Acyl- and Silyl- Resveratrol Derivatives as Retinoprotective Agents: Piceid Octanoate Notably Delays Photoreceptor Degeneration in a Retinitis Pigmentosa Mouse Model
de réir: Lourdes Valdés-Sánchez, et al.
Foilsithe / Cruthaithe: (2024-11-01)

Single-cell RNA sequencing of the retina in a model of retinitis pigmentosa reveals early responses to degeneration in rods and cones
de réir: Duygu Karademir, et al.
Foilsithe / Cruthaithe: (2022-04-01)

Retinitis pigmentosa and congenital toxoplasmosis: A rare coexistence
de réir: Chhabra Manpreet, et al.
Foilsithe / Cruthaithe: (2007-01-01)

Mitochondrial Transplantation Moderately Ameliorates Retinal Degeneration in Royal College of Surgeons Rats
de réir: Shih-Fang Wu, et al.
Foilsithe / Cruthaithe: (2022-11-01)

Pupil size following dark adaptation in patients with retinitis pigmentosa
de réir: A. Berezovsky, et al.
Foilsithe / Cruthaithe: (2001-08-01)

Patient-specific iPSC-derived photoreceptor precursor cells as a means to investigate retinitis pigmentosa
de réir: Budd A Tucker, et al.
Foilsithe / Cruthaithe: (2013-08-01)

Microglial phagocytosis of living photoreceptors contributes to inherited retinal degeneration
de réir: Lian Zhao, et al.
Foilsithe / Cruthaithe: (2015-07-01)

Comparison of structural progression between ciliopathy and non-ciliopathy associated with autosomal recessive retinitis pigmentosa
de réir: Vitor K. L. Takahashi, et al.
Foilsithe / Cruthaithe: (2019-08-01)

Schizophrenia and Retinitis Pigmentosa – A case report
de réir: Narayan R Mutalik, et al.
Foilsithe / Cruthaithe: (2016-01-01)

Nonclinical Safety Evaluation of scAAV8-RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa
de réir: Timothy K. MacLachlan, et al.
Foilsithe / Cruthaithe: (2018-03-01)

Ocular Toxoplasmosis Associated with Unilateral Pigmentary Retinopathy That May Mimic Retinitis Pigmentosa: Diagnostic Dilemmas
de réir: Izabella Karska-Basta, et al.
Foilsithe / Cruthaithe: (2021-08-01)

Prothrombin time in retinitis pigmentosa
de réir: Vinchurkar Manisha
Foilsithe / Cruthaithe: (1998-01-01)

Concurrent retinitis pigmentosa and pigmented paravenous retinochoroidal atrophy phenotypes in the same patient
de réir: Dhanashree Ratra, et al.
Foilsithe / Cruthaithe: (2016-01-01)

Optimizing an AAV-CRISPR system for retinal gene therapy
de réir: Stevanovic, M
Foilsithe / Cruthaithe: (2019)

PATTERN ELECTRORETINOGRAPHY IN RELATION TO KINETIC AND STATIC PERIMETRY AND VISUAL ACUITY IN RETINITIS PIGMENTOSA
de réir: Petra Popović, et al.
Foilsithe / Cruthaithe: (2002-12-01)

Disease modeling and pharmacological rescue of autosomal dominant retinitis pigmentosa associated with RHO copy number variation
de réir: Sangeetha Kandoi, et al.
Foilsithe / Cruthaithe: (2024-04-01)

Proinflammatory Pathways Are Activated in the Human Q344X Rhodopsin Knock-In Mouse Model of Retinitis Pigmentosa
de réir: T.J. Hollingsworth, et al.
Foilsithe / Cruthaithe: (2021-08-01)