RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Gene editing stands for the methods to precisely make changes to a specific nucleic acid
sequence. With the recent development of the clustered regularly interspaced short …

CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Throughout its 40-year history, the field of gene therapy has been marked by many
transitions. It has seen great strides in combating human disease, has given hope to patients …

Gene editing that makes target gene modification in the genome by deletion or addition has
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …

The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Because of their small size, the recently developed CRISPR-Cas12f nucleases can be
effectively packaged into adeno-associated viruses for gene therapy. However, a systematic …

Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …

Recombinant Adeno-associated virus (rAAV) is one of the main delivery vectors for gene
therapy. To assess immunogenicity, toxicity, and features of AAV gene therapy in clinical …