Results 121 to 130 of about 1,632,775 (273)
CRISPR-Cas9–based treatment of myocilin-associated glaucoma [PDF]
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
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
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]
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
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
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
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]
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]
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