The application and progression of CRISPR/Cas9 technology in ophthalmological diseases
X Hu, B Zhang, X Li, M Li, Y Wang, H Dan, J Zhou… - Eye, 2023 - nature.com
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-
associated nuclease (Cas) system is an adaptive immune defence system that has gradually …
associated nuclease (Cas) system is an adaptive immune defence system that has gradually …
Genome-editing strategies for treating human retinal degenerations
J Quinn, A Musa, A Kantor, ME McClements… - Human Gene …, 2021 - liebertpub.com
Inherited retinal degenerations (IRDs) are a leading cause of blindness. Although gene-
supplementation therapies have been developed, they are only available for a small …
supplementation therapies have been developed, they are only available for a small …
Genome editing, a superior therapy for inherited retinal diseases
Gene augmentation and genome editing are promising strategies for the treatment of
monogenic inherited retinal diseases. Although gene augmentation treatments are …
monogenic inherited retinal diseases. Although gene augmentation treatments are …
Increasing the targeting scope of CRISPR base editing system beyond NGG
SY Yu, A Birkenshaw, T Thomson, T Carlaw… - The CRISPR …, 2022 - liebertpub.com
Genome editing provides a new therapeutic strategy to cure genetic diseases. The recently
developed CRISPR-Cas9 base editing technology has shown great potential to repair the …
developed CRISPR-Cas9 base editing technology has shown great potential to repair the …
CRISPR genome engineering for retinal diseases
Novel gene therapy treatments for inherited retinal diseases have been at the forefront of
translational medicine over the past couple of decades. Since the discovery of CRISPR …
translational medicine over the past couple of decades. Since the discovery of CRISPR …
Advances in engineering CRISPR-Cas9 as a molecular Swiss Army knife
GA Meaker, EJ Hair, TE Gorochowski - Synthetic Biology, 2020 - academic.oup.com
The RNA-guided endonuclease system CRISPR-Cas9 has been extensively modified since
its discovery, allowing its capabilities to extend far beyond double-stranded cleavage to high …
its discovery, allowing its capabilities to extend far beyond double-stranded cleavage to high …
New technologies to study functional genomics of age-related macular degeneration
T Nguyen, D Urrutia-Cabrera, RHC Liou… - Frontiers in Cell and …, 2021 - frontiersin.org
Age-related macular degeneration (AMD) is the most common cause of irreversible vision
loss in people over 50 years old in developed countries. Currently, we still lack a …
loss in people over 50 years old in developed countries. Currently, we still lack a …
CRISPR Cas9 based genome editing in inherited retinal dystrophies
M Bansal, S Acharya, S Sharma, R Phutela… - Ophthalmic …, 2021 - Taylor & Francis
Background Precision genome engineering, with targeted therapy towards patient-specific
mutations is predicted to be the future of personalized medicine. Ophthalmology is in the …
mutations is predicted to be the future of personalized medicine. Ophthalmology is in the …
Human pluripotent stem cells for the modelling of retinal pigment epithelium homeostasis and disease: A review
Human pluripotent stem cells (hPSCs), which include induced pluripotent stem cells and
embryonic stem cells, are powerful tools for studying human development, physiology and …
embryonic stem cells, are powerful tools for studying human development, physiology and …
Development of reporter mouse models to evaluate and optimize crispr/cas9 base editing therapeutics
S Yu - 2022 - open.library.ubc.ca
Genetic diseases are a leading cause of death and disability worldwide with immense
economic and societal burdens. However, currently fewer than 5% of genetic diseases have …
economic and societal burdens. However, currently fewer than 5% of genetic diseases have …