Genome engineering via targeted nucleases, specifically CRISPR-Cas9, has revolutionized
the field of gene therapy research, providing a potential treatment for diseases of the blood …

Inborn errors of immunity (IEIs) are a group of inherited disorders caused by mutations in the
protein-coding genes involved in innate and/or adaptive immunity. Hematopoietic stem cell …

Severe combined immunodeficiency (SCID) is a group of disorders caused by mutations in
genes involved in the process of lymphocyte maturation and function. CRISPR-Cas9 gene …

Genome editing involves precise modification of specific nucleotides in the genome using
nucleases like CRISPR/Cas, ZFN, or TALEN, leading to increased efficiency of homologous …

With the introduction of precision genome editing using CRISPR–Cas9 technology, we have
entered a new era of genetic engineering and gene therapy. With RNA-guided …

Ex vivo gene correction of hematopoietic stem and progenitor cells (HSPCs) has emerged
as a promising therapeutic approach for treatment of inherited human blood disorders. Use …

The advent of next-generation genome engineering tools like CRISPR-Cas9 has
transformed the field of gene therapy, rendering targeted treatment for several incurable …

Clustered regularly interspaced short palindromic repeat-Cas9 (CRISPR/Cas9), derived
from bacterial and archean immune systems, has received much attention from the scientific …

Primary immunodeficiency diseases (PIDs) encompass a range of diseases due to
mutations in genes that are critical for immunity. Haploinsufficiency and gain-of-function …

Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …