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 …

RNA‐based therapeutics have the potential to revolutionize the treatment and prevention of
human diseases. While early research faced setbacks, it established the basis for …

T cell receptor-engineered T cell (TCR-T) therapy is free from the limit of surface antigen
expression of the target cells, which is a potential cellular immunotherapy for cancer …

Regulatory T cells are critical for maintaining immune tolerance. Recent studies have
confirmed their therapeutic suppressive potential to modulate immune responses in organ …

CRISPR offers new hope for many patients and promises to transform the way we think of
future therapies. Ensuring safety of CRISPR therapeutics is a top priority for clinical …

Biomedical researchers have subsequently been inspired the development of new
approaches for precisely changing an organism's genomic DNA in order to investigate …

Genome editing has been a transformative force in the life sciences and human medicine,
offering unprecedented opportunities to dissect complex biological processes and treat the …

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by
mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which …

Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Abnormal gene expression level or expression of genes containing deleterious mutations
are two of the main determinants which lead to genetic disease. To obtain a therapeutic …