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Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System.

Abigail McElroyMiguel Sena-EstevesMotahareh ArjomandnejadAllison M KeelerHeather L Gray-Edwards
Published in: Human gene therapy (2022)
Adeno-associated virus (AAV)-mediated gene therapies have provided promising treatments for numerous neurological disorders. Redosing of AAV to the central nervous system (CNS) is an attractive research area due to both the somewhat immunologically privileged status of the CNS as well as the possibility of reduced glial transgene expression over time following a single injection. Continued study of the immune responses to both intraparenchymal and intra-CSF delivery of AAV mediated gene therapies, as well as the continued study of immunosuppressive regimens, could allow for eventual redosing in patients.
Keyphrases
  • gene therapy
  • immune response
  • end stage renal disease
  • cerebrospinal fluid
  • newly diagnosed
  • ejection fraction
  • chronic kidney disease
  • copy number
  • gene expression
  • brain injury
  • dna methylation
  • transcription factor