Gene therapy is emerging as a powerful tool to modulate abnormal gene expression, a
hallmark of most CNS disorders. The transformative potentials of recently approved gene …

Huntington's disease (HD) is a paradigm of a genetic neurodegenerative disorder
characterized by the expansion of CAG repeats in the HTT gene. This extensive review …

In Huntington's disease (HD), striatal projection neurons (SPNs) degenerate during midlife;
the core biological question involves how the disease-causing DNA repeat (CAG) n in the …

In this edition of the Huntington's Disease Clinical Trials Update, we expand on the ongoing
program from VICO Therapeutics and on the recently terminated VIBRANT-HD clinical trials …

Despite investment of billions of research dollars, neurodegenerative diseases have been
largely resistant to therapeutics development because of incomplete understanding of …

Abstract Purpose of Review Huntington's disease (HD) is an autosomal-dominant disorder
caused by a pathological expansion of a trinucleotide repeat (CAG) on exon 1 of the …

In this review, we summarize preclinical and clinical trials investigating innovative
neuromodulatory approaches for Parkinson disease (PD) motor symptom management. We …

Neurological and neuromuscular diseases resulting from familial, sporadic, or de novo
mutations have devasting personal, familial, and societal impacts. As the initial product of …

Oligonucleotide therapeutics (ASOs and siRNAs) have been explored for modulation of
gene expression in the central nervous system (CNS), with several drugs approved and …

Rapid advances in biological knowledge and technological innovation have greatly
advanced the fields of stem cell and gene therapies to combat a broad spectrum of …