Engineering adeno-associated virus vectors for gene therapy
C Li, RJ Samulski - Nature Reviews Genetics, 2020 - nature.com
Adeno-associated virus (AAV) vector-mediated gene delivery was recently approved for the
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …
treatment of inherited blindness and spinal muscular atrophy, and long-term therapeutic …
Gene therapy leaves a vicious cycle
R Goswami, G Subramanian, L Silayeva… - Frontiers in …, 2019 - frontiersin.org
The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …
proteins that drive all biological processes necessary for normal life and death. Though the …
A positively tuned voltage indicator for extended electrical recordings in the brain
Genetically encoded voltage indicators (GEVIs) enable optical recording of electrical signals
in the brain, providing subthreshold sensitivity and temporal resolution not possible with …
in the brain, providing subthreshold sensitivity and temporal resolution not possible with …
Deep diversification of an AAV capsid protein by machine learning
Modern experimental technologies can assay large numbers of biological sequences, but
engineered protein libraries rarely exceed the sequence diversity of natural protein families …
engineered protein libraries rarely exceed the sequence diversity of natural protein families …
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
Loss of perivascular aquaporin-4 localization impairs glymphatic exchange and promotes amyloid β plaque formation in mice
Background Slowed clearance of amyloid β (Aβ) is believed to underlie the development of
Aβ plaques that characterize Alzheimer's disease (AD). Aβ is cleared in part by the …
Aβ plaques that characterize Alzheimer's disease (AD). Aβ is cleared in part by the …
Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems
Adeno-associated viruses (AAVs) are commonly used for in vivo gene transfer.
Nevertheless, AAVs that provide efficient transduction across specific organs or cell …
Nevertheless, AAVs that provide efficient transduction across specific organs or cell …
[HTML][HTML] Ultrafast two-photon imaging of a high-gain voltage indicator in awake behaving mice
Optical interrogation of voltage in deep brain locations with cellular resolution would be
immensely useful for understanding how neuronal circuits process information. Here, we …
immensely useful for understanding how neuronal circuits process information. Here, we …
Immune responses to retinal gene therapy using adeno-associated viral vectors–Implications for treatment success and safety
K Bucher, E Rodríguez-Bocanegra… - Progress in retinal and …, 2021 - Elsevier
Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …
Gene therapy for neurological disorders: progress and prospects
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …