Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Cell-type-specific expression of molecular tools and sensors is critical to construct circuit
diagrams and to investigate the activity and function of neurons within the nervous system …

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of
hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene …

Gene therapy is rapidly emerging as a powerful therapeutic strategy for a wide range of
neurodegenerative disorders, including Alzheimer's disease (AD), Parkinson's disease (PD) …

Gene silencing with virally delivered shRNA represents a promising approach for treatment
of inherited neurodegenerative disorders. In the present study we develop a subpial …

Traumatic spinal cord injury (SCI) elicits an acute inflammatory response which comprises
numerous cell populations. It is driven by the immediate response of macrophages and …

The mRNA transcript of the human STMN2 gene, encoding for stathmin-2 protein (also
called SCG10), is profoundly impacted by TAR DNA-binding protein 43 (TDP-43) loss of …

Introduction: Amyotrophic lateral sclerosis (ALS) is a progressive and incurable
neurodegenerative disorder that targets upper and lower motor neurons and leads to fatal …

Introduction: Adeno-associated viral (AAV) vector-mediated gene delivery to the spinal cord
has finally entered the pathway towards regulatory approval. Phase 1 clinical trials using …

As the need for nasal, ocular, spinal, and articular therapeutic compounds increases,
toxicology assessments of drugs administered via these routes play an important role in …