The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

Gene-editing technologies, which include the CRISPR–Cas nucleases 1, 2, 3 and CRISPR
base editors 4, 5, have the potential to permanently modify disease-causing genes in …

Sickle cell disease (SCD) is one of the most common hemoglobinopathies. Due to its high
prevalence, with about 20 million affected individuals worldwide, the development of novel …

Prime editors have a broad range of potential research and clinical applications. However,
methods to delineate their genome-wide editing activities have generally relied on indirect …

Summary The c. 1222C> T (p. Arg408Trp) variant in the phenylalanine hydroxylase gene
(PAH) is the most frequent cause of phenylketonuria (PKU), the most common inborn error of …

Phenylketonuria (PKU), an autosomal recessive disorder caused by pathogenic variants in
the phenylalanine hydroxylase (PAH) gene, results in the accumulation of blood …

Presenilin 1 (PS1) is a central component of γ-secretase, an enzymatic complex involved in
the generation of the amyloid-β (Aβ) peptide that deposits as plaques in the Alzheimer's …

Prime editors (PEs) are powerful tools that widen the possibilities for sequence modifications
during genome editing. Although methods based on the analysis of Cas9 nuclease or …

The advancement of CRISPR-based gene editing tools into biotherapeutics offers the
potential for cures to genetic disorders and for new treatment paradigms for even common …

Alpha-1 antitrypsin deficiency (AATD) is a rare autosomal codominant disease caused by
mutations within the SERPINA1 gene. The most prevalent variant in patients is PiZ …