Recent progress and considerations for AAV gene therapies targeting the central nervous system
EA Lykken, C Shyng, RJ Edwards, A Rozenberg… - Journal of …, 2018 - Springer
Background Neurodevelopmental disorders, as a class of diseases, have been particularly
difficult to treat even when the underlying cause (s), such as genetic alterations, are …
difficult to treat even when the underlying cause (s), such as genetic alterations, are …
Adeno-associated virus vectors and neurological gene therapy
DS Ojala, DP Amara, DV Schaffer - The Neuroscientist, 2015 - journals.sagepub.com
Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …
[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges
L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …
central nervous system (CNS) gene therapy. AAV has already shown promising results in …
Adeno-associated virus (AAV) vectors in the CNS
TJ McCown - Current gene therapy, 2011 - ingentaconnect.com
Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this
vector system an excellent choice for both CNS gene therapy and basic neurobiological …
vector system an excellent choice for both CNS gene therapy and basic neurobiological …
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 …
AAV-based in vivo gene therapy for neurological disorders
Q Ling, JA Herstine, A Bradbury, SJ Gray - Nature Reviews Drug …, 2023 - nature.com
Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …
treatment of neurological disorders. Among the available delivery vehicles, adeno …
Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders
L Marrone, PM Marchi, M Azzouz - Expert Opinion on Biological …, 2022 - Taylor & Francis
Introduction Gene therapy provides the exciting opportunity of a curative single treatment for
devastating diseases, eradicating the need for chronic medication. Adeno-associated …
devastating diseases, eradicating the need for chronic medication. Adeno-associated …
Adeno-associated virus (AAV) vectors in the CNS
TJ McCown - Current gene therapy, 2005 - ingentaconnect.com
Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this
vector system an excellent choice for both CNS gene therapy and basic neurobiological …
vector system an excellent choice for both CNS gene therapy and basic neurobiological …
Delivering AAV to the central nervous and sensory systems
As gene therapy enters mainstream medicine, it is more important than ever to have a grasp
of exactly how to leverage it for maximum benefit. The development of new targeting …
of exactly how to leverage it for maximum benefit. The development of new targeting …
Therapeutic AAV gene transfer to the nervous system: a clinical reality
E Hudry, LH Vandenberghe - Neuron, 2019 - cell.com
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …
considered for rare inherited disorders, gene therapy may open treatment opportunities for …