Adeno-associated viruses (AAVs) are being increasingly used as gene therapy vectors in
clinical studies especially targeting central nervous system (CNS) disorders …

Current treatments for chronic pain rely largely on opioids despite their substantial side
effects and risk of addiction. Genetic studies have identified in humans key targets pivotal to …

The identification of the most efficient method for whole central nervous system targeting that
is translatable to humans and the safest route of adeno-associated virus (AAV) …

Objectives Spinal muscular atrophy (SMA) is caused by reduced levels of survival motor
neuron (SMN) protein, which results in motoneuron loss. Therapeutic strategies to increase …

Many studies have demonstrated that adeno-associated virus serotype 9 (AAV9) transduces
astrocytes and neurons when infused into rat or nonhuman primate (NHP) brain. We …

Mucopolysaccharidoses (MPS) are inborn errors of metabolism produced by a deficiency of
one of the enzymes involved in the degradation of glycosaminoglycans (GAGs). Although …

Spastic paraplegia 50 (SPG50) is an ultrarare childhood-onset neurological disorder caused
by biallelic loss-of-function variants in the AP4M1 gene. SPG50 is characterized by …

Adeno-associated viral vectors (AAVs) have demonstrated potential in applications for
neurologic disorders, and the discovery that some AAVs can cross the blood-brain barrier …

Gene transfer is an increasingly utilized approach for research and clinical applications
involving the central nervous system (CNS). Vectors for gene transfer can be as simple as …

The past several years have witnessed significant advances in the development of
therapeutic gene delivery for neurological disorders of the central nervous system (CNS). In …