In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …

With the advent of recombinant DNA technology in the 1970s, the idea of using gene
therapies to treat human genetic diseases captured the interest and imagination of scientists …

Cytosine base editors (CBEs) enable programmable genomic C· G-to-T· A transition
mutations and typically comprise a modified CRISPR–Cas enzyme, a naturally occurring …

Sickle cell disease (SCD), a distinctive and often overlooked illness in the 21st century, is a
congenital blood disorder characterized by considerable phenotypic diversity. It comprises a …

Hepatitis B virus (HBV) remains a significant cause of mortality and morbidity worldwide,
since chronic HBV infection is associated with elevated risk of cirrhosis and hepatocellular …

The clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-
associated protein 9 (Cas9)(CRISPR/Cas9) system is a powerful tool that enables precise …

Gene therapies have conspicuously bloomed in recent years as evidenced by the
increasing number of cell-, gene-, and oligo-based approved therapies. These therapies …

In recent years, clustered regularly interspaced short palindromic repeats (CRISPRs) and
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …

Ganglioside-monosialic acid (GM1) gangliosidosis, a rare autosomal recessive disorder, is
frequently caused by deleterious single nucleotide variants (SNVs) in GLB1 gene. These …

High molecular weight actives and cell-based therapy have the potential to revolutionize the
prophylaxis and therapy of severe diseases. Yet, the size and nature of the agents–proteins …