Neurodegenerative diseases (NDs) such as Alzheimer disease (AD), Parkinson disease
(PD), and Huntington disease (HD) represent a major socio-economic challenge in view of …

Huntington's disease is caused by an abnormally expanded CAG repeat expansion in the
HTT gene, which confers a predominant toxic gain of function in the mutant huntingtin …

Conspectus The discovery of CRISPR/Cas has revolutionized the field of genome editing.
CRIPSR/Cas components are part of the bacterial immune system and are able to induce …

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

Huntington's disease (HD) is a neurodegenerative disorder caused by a CAG expansion in
the HD gene. The disease is characterized by neurodegeneration, particularly in the striatum …

Genome editing reemerged in 2012 with the development of CRISPR/Cas9 technology,
which is a genetic manipulation tool derived from the defense system of certain bacteria …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

Genetic diseases seriously threaten human health and have always been one of the
refractory conditions facing humanity. Currently, gene therapy drugs such as siRNA, shRNA …

Smithies et al.(1985) and Jasin and colleagues (1994) provided proof of concept that
homologous recombination (HR) could be applied to the treatment of human disease and …

Huntington's disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative
disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin …