Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in develo** clinically …

Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …

The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …

Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …

CRISPR/Cas genome editing is a simple, cost effective, and highly specific technique for
introducing genetic variations. In mammalian cells, CRISPR/Cas can facilitate non …

Rare genetic diseases, often referred to as orphan diseases due to their low prevalence and
limited treatment options, have long posed significant challenges to our medical system. In …

Since its mechanism discovery in 2012 and the first application for mammalian genome
editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …

Based on the high frequency of concurrent adenomatous polyposis coli (APC) and KRAS
mutations and their strong cooperative interaction in human colorectal cancer (CRC) …

Targeted nucleases are powerful genomic tools to precisely change the target genome of
living cells, controlling functional genes with high exactness. The clustered regularly …

Promising improvements in the field of transcript therapeutics have clearly enhanced the
potential of mRNA as a new pillar for protein replacement therapies. Synthetic mRNAs are …