Over recent decades, therapeutic proteins have had widespread success in treating a
myriad of diseases. Glycosylation, a near universal feature of this class of drugs, is a critical …

In contrast to the standard enzyme-replacement therapy, administered from once per 7–14
days to 2–3 times a week in patients with severe hemophilia B, as a result of a single …

The engineering construction of the liver has attracted enormous attention. Organoids, as
emerging miniature three-dimensional cultivation units, hold significant potential in the …

Recombinant adeno-associated virus (rAAV) is one of the prominent gene delivery vehicles
that has opened promising opportunities for novel gene therapeutic approaches. However …

Impressive achievements in clinical trials to treat hemophilia establish a milestone in the
development of gene therapy. It highlights the significance of AAV-mediated gene delivery to …

Suicide gene therapy is a promising strategy for the potential treatment of hepatocellular
carcinoma (HCC). However, the lack of high transduction efficiency and targeted vectors in …

Hemophilic arthropathy (HA) due to repeated bleeding into the joint cavity is a major cause
of morbidity in patients with hemophilia. The molecular mechanisms contributing to this …

Inherited genetic disorders are progressive in nature and lead to organ dysfunction or death
in severe cases. At present, there are no permanent treatment options for> 95% of inherited …

Stroke is a major cause of morbidity and mortality worldwide. In the early stages of stroke,
irreversible damage to neurons leads to high mortality and disability rates in patients …

Dear Editor, Anti-CD19/CD3 bispecific T-cell engagers (CD19BiTE) have shown promising
efficacy in patients with relapsed or refractory (r/r) B-cell acute lymphoblastic leukemia (B …