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

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …

Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic
genome editing but are severely constrained by cargo limits. Simultaneous delivery of …

Gene therapy is poised to revolutionize modern medicine, with seemingly unlimited potential
for treating and curing genetic disorders. For otherwise incurable indications, including most …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …

Gene editing-based therapeutic strategies grant the power to override cell machinery and
alter faulty genes contributing to disease development like cancer. Nowadays, the principal …

Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …

CRISPR/Cas9 is an effective gene editing tool with broad applications for the prevention or
treatment of numerous diseases. It depends on CRISPR (clustered regularly interspaced …

Autologous hematopoietic stem cell (HSC)-targeted gene therapy provides a one-time cure
for various genetic diseases including sickle cell disease (SCD) and β-thalassemia. SCD is …

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