Alzheimer's disease (AD) and Parkinson's disease (PD) are the most prevalent
neurodegenerative diseases, affecting millions and costing billions each year in the United …

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 …

At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …

Adeno-associated virus (AAV) vectors have shown promising results in preclinical models,
but the genomic consequences of transduction with AAV vectors encoding CRISPR-Cas …

Manganese is an essential trace metal. The dysregulation of manganese seen in a broad
spectrum of neurological disorders reflects its importance in brain development and key …

CRISPR genome editing can potentially treat the root causes of many genetic diseases,
including central nervous system (CNS) disorders. However, the promise of brain‐targeted …

Since most dominant human mutations are single nucleotide substitutions,, we explored
gene editing strategies to disrupt dominant mutations efficiently and selectively without …

Background Alzheimer's disease (AD) is an insidious, irreversible, and progressive
neurodegenerative health condition manifesting as cognitive deficits and amyloid beta (Aβ) …

Mitochondria play a vital role in the nervous system, as they are responsible for generating
energy in the form of ATP and regulating cellular processes such as calcium (Ca 2+) …

CRISPR/Cas9-based genome editing has quickly emerged as a powerful breakthrough
technology for use in diverse settings across biomedical research and therapeutic …