Systemic delivery of self-complementary (sc) adeno-associated-virus vector of serotype 9
(AAV9) was recently shown to provide robust and widespread gene transfer to the central …

Adeno-associated virus (AAV) is frequently used for gene transfer into the central nervous
system (CNS). Similar to adenovirus and rabies virus, AAV can be taken up by axons and …

Recombinant adeno-associated virus serotype 2 (rAAV2) vectors have shown promise as
therapeutic agents for neurologic disorders. However, intracerebral administration of this …

Recombinant viruses allow for targeted transgene expression in specific cell populations
throughout the nervous system. The adeno-associated virus (AAV) is among the most …

The development of gene therapies for the treatment of diseases of the central nervous
system has been hindered by the limited availability of adeno-associated viruses (AAVs) that …

Some recombinant adeno-associated viruses (rAAVs) can cross the neonatal blood–brain
barrier (BBB) and efficiently transduce cells of the central nervous system (CNS). However …

Transduction of the primate cortex with adeno-associated virus (AAV)-based gene therapy
vectors has been challenging, because of the large size of the cortex. We report that a single …

Adeno-associated virus (AAV)-based gene therapies, exemplified by the approved therapy
for spinal muscular atrophy, have the potential to deliver disease-course-altering treatments …

Targeting lower motor neurons (LMNs) for gene delivery could be useful for disorders such
as spinal muscular atrophy and amyotrophic lateral sclerosis. LMNs reside in the ventral …

Recombinant adeno-associated viruses (rAAV) are highly efficient vectors for gene delivery
into the central nervous system (CNS). However, host inflammatory and immune responses …