Results 121 to 130 of about 1,632,775 (273)

CRISPR-Cas9–based treatment of myocilin-associated glaucoma [PDF]

open access: yes, 2018
Primary open-angle glaucoma (POAG) is a leading cause of irreversible vision loss worldwide, with elevated intraocular pressure (IOP) a major risk factor. Myocilin (MYOC) dominant gain-of-function mutations have been reported in ∼4% of POAG cases.
Bugge, Kevin   +11 more
core   +1 more source

Comparative evaluation of four exome enrichment solutions in 2024: Agilent, Roche, Vazyme and Nanodigmbio

open access: yesBMC Genomics
Whole exome sequencing (WES) is essential for identifying genetic variants linked to diseases. This study compares available to date four exome enrichment kits: Agilent SureSelect Human All Exon v8, Roche KAPA HyperExome, Vazyme VAHTS Target Capture Core
Vera Belova   +12 more
doaj   +1 more source

Adenoviral Vectors for Gene Therapy of Hereditary Diseases

open access: yesBiology
Adenoviral vectors (AdVs) are effective vectors for gene therapy due to their broad tropism, high capacity, and high transduction efficiency, which makes them actively used as oncolytic vectors and for creating vector vaccines.
Anna Muravyeva, Svetlana Smirnikhina
doaj   +1 more source

Potential Application of the CRISPR/Cas9 System against Herpesvirus Infections. [PDF]

open access: yes, 2018
The CRISPR/Cas9 system has been applied in the genome editing and disruption of latent infections for herpesviruses such as the herpes simplex virus, Epstein⁻Barr virus, cytomegalovirus, and Kaposi's sarcoma-associated herpesvirus.
Chen, Yuan-Chuan   +3 more
core   +2 more sources

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

open access: yesCells, 2020
Gene editing that makes target gene modification in the genome by deletion or addition has revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 emerged as a substantial tool due to its simplicity ...
Muhammad Naeem   +3 more
semanticscholar   +1 more source

Structural variation of types IV-A1- and IV-A3-mediated CRISPR interference

open access: yesNature Communications
CRISPR-Cas mediated DNA-interference typically relies on sequence-specific binding and nucleolytic degradation of foreign genetic material. Type IV-A CRISPR-Cas systems diverge from this general mechanism, using a nuclease-independent interference ...
R. Čepaitė   +14 more
doaj   +1 more source

The CRISPR tool kit for genome editing and beyond

open access: yesNature Communications, 2018
CRISPR is becoming an indispensable tool in biological research. Once known as the bacterial immune system against invading viruses, the programmable capacity of the Cas9 enzyme is now revolutionizing diverse fields of medical research, biotechnology ...
Mazhar Adli
semanticscholar   +1 more source

The genome editing revolution: review [PDF]

open access: yesJournal of Genetic Engineering and Biotechnology, 2020
Development of efficient strategies has always been one of the great perspectives for biotechnologists. During the last decade, genome editing of different organisms has been a fast advancing field and therefore has received a lot of attention from various researchers comprehensively reviewing latest achievements and offering opinions on future ...
openaire   +4 more sources

CAUSEL: an epigenome- and genome-editing pipeline for establishing function of noncoding GWAS variants [PDF]

open access: yes, 2015
The vast majority of disease-associated single nucleotide polymorphisms (SNPs) mapped by genome-wide association studies (GWAS) are located in the non-protein coding genome, but establishing the functional and mechanistic roles of these sequence variants
Christopher Haiman   +19 more
core   +3 more sources

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