The development of high efficiency, central nervous system (CNS) targeting AAV-based
gene therapies is necessary to address challenges in both pre-clinical and clinical …

Using single-stranded adeno-associated virus serotype 9 (ssAAV9) vectors containing the
neuron-specific synapsin-I promoter, we examined whether different administration routes …

Adeno-associated virus (AAV) is the most common vector for clinical gene therapy of the
CNS. This popularity originates from a high safety record and the longevity of transgene …

AAV9 has emerged as an efficient adeno-associated virus (AAV) serotype for gene transfer
to the central nervous system. We have used this technique to study aspects of amyotrophic …

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 viral (AAV) vectors are an established and safe gene delivery tool to
target the nervous system. However, the payload capacity of< 4.9 kb limits the transfer of …

Cell-selective gene expression comprises a critical element of many adeno-associated virus
(AAV) vector-based gene therapies, and to date achieving this goal has focused on AAV …

Gene therapy for motor neuron diseases requires efficient gene delivery to motor neurons
(MNs) throughout the spinal cord and brainstem. The present study compared adeno …

The adult mammalian brain entails a reservoir of neural stem cells (NSCs) generating glial
cells and neurons. However, NSCs become increasingly quiescent with age, which hampers …

Adeno-associated virus (AAV) vector, an excellent gene therapy vector, has been widely
used in the treatment of various central nervous system (CNS) diseases. Due to the …