Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with
no or limited treatment options. This is the case for hundreds of monogenic metabolic …

Gene therapy encompasses the transfer of exogenous genetic materials into the patient's
target cells to treat or prevent diseases. Nevertheless, the transfer of genetic material into …

Impressive therapeutic advances have been possible through the advent of zinc-finger
nucleases and transcription activator-like effector nucleases. However, discovery of the …

As a cytosol ubiquitin ligase and antibody receptor, Tripartite motif-containing 21 (TRIM21)
has been reported to mediate the restriction of hepatitis B virus (HBV) through an HBx …

One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia
A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a …

The overexpression of SARS-CoV-2 primary receptors and co-receptors (ACE2, TMPRSS2,
FURIN, and CD147) enhance the likeliness of SARS-CoV-2 infection. The genes for same …

Hepatic gene therapy by delivering non-integrating therapeutic vectors in newborns remains
challenging due to the risk of dilution and loss of efficacy in the growing liver. Previously we …

Osteoporosis is a metabolic bone disease that impairs bone mineral density,
microarchitecture, and strength. It requires continuous management, and further research …

Compound heterozygous recessive or polygenic diseases could be addressed through
gene correction of multiple alleles. However, targeting of multiple alleles using genome …