Matches in SemOpenAlex for { <https://semopenalex.org/work/W4313200216> ?p ?o ?g. }
Showing items 1 to 65 of
65
with 100 items per page.
- W4313200216 endingPage "4" @default.
- W4313200216 startingPage "1" @default.
- W4313200216 abstract "Gene therapy using adeno-associated virus (AAV) vectors is rapidly developing. AAV2 vectors are injected directly into the brain by stereotaxic brain surgery and have shown good results in Parkinson's disease and aromatic L-amino acid decarboxylase deficiency. An AAV9 vector-derived product for spinal muscular atrophy type 1 has been administered to more than 2000 patients worldwide. AAV vectors, which cross the blood-brain barrier, have been applied to the development of gene therapy for various neurological diseases. We are preparing for clinical trials for glucose transporter I deficiency, Niemann-Pick disease type C, GM2 gangliosidosis, sporadic amyotrophic lateral sclerosis, and spinocerebellar ataxia type 1. AAV.GT5, which can efficiently transfer genes to human hepatocytes, is planned for clinical trials for ornithine transcarbamylase deficiency and hemophilia B. Wildtype AAVs are considered to be nonpathogenic. The genome of the AAV vector resides in the episome and is rarely integrated into chromosomes, making the vectors highly safe. On the other hand, adverse events that require further attention are reported. Four patients died in a clinical trial of X-linked myotubular myopathy in which a high dose of AAV vector was administered. Clonal proliferation of hepatocytes was observed in a hemophilia canine long after gene therapy. Neuronal loss was observed in the dorsal root ganglia of primates treated with various AAV vectors." @default.
- W4313200216 created "2023-01-06" @default.
- W4313200216 creator A5070966533 @default.
- W4313200216 date "2022-01-01" @default.
- W4313200216 modified "2023-10-16" @default.
- W4313200216 title "AAV vector-based gene therapy: current status and challenges" @default.
- W4313200216 doi "https://doi.org/10.1254/jpssuppl.96.0_1-b-s12-4" @default.
- W4313200216 hasPublicationYear "2022" @default.
- W4313200216 type Work @default.
- W4313200216 citedByCount "0" @default.
- W4313200216 crossrefType "journal-article" @default.
- W4313200216 hasAuthorship W4313200216A5070966533 @default.
- W4313200216 hasBestOaLocation W43132002161 @default.
- W4313200216 hasConcept C104317684 @default.
- W4313200216 hasConcept C111599444 @default.
- W4313200216 hasConcept C142724271 @default.
- W4313200216 hasConcept C2777468819 @default.
- W4313200216 hasConcept C2778107364 @default.
- W4313200216 hasConcept C2778558090 @default.
- W4313200216 hasConcept C2779134260 @default.
- W4313200216 hasConcept C2780469793 @default.
- W4313200216 hasConcept C32470452 @default.
- W4313200216 hasConcept C40767141 @default.
- W4313200216 hasConcept C515207424 @default.
- W4313200216 hasConcept C54355233 @default.
- W4313200216 hasConcept C71924100 @default.
- W4313200216 hasConcept C77061444 @default.
- W4313200216 hasConcept C86803240 @default.
- W4313200216 hasConcept C92087593 @default.
- W4313200216 hasConceptScore W4313200216C104317684 @default.
- W4313200216 hasConceptScore W4313200216C111599444 @default.
- W4313200216 hasConceptScore W4313200216C142724271 @default.
- W4313200216 hasConceptScore W4313200216C2777468819 @default.
- W4313200216 hasConceptScore W4313200216C2778107364 @default.
- W4313200216 hasConceptScore W4313200216C2778558090 @default.
- W4313200216 hasConceptScore W4313200216C2779134260 @default.
- W4313200216 hasConceptScore W4313200216C2780469793 @default.
- W4313200216 hasConceptScore W4313200216C32470452 @default.
- W4313200216 hasConceptScore W4313200216C40767141 @default.
- W4313200216 hasConceptScore W4313200216C515207424 @default.
- W4313200216 hasConceptScore W4313200216C54355233 @default.
- W4313200216 hasConceptScore W4313200216C71924100 @default.
- W4313200216 hasConceptScore W4313200216C77061444 @default.
- W4313200216 hasConceptScore W4313200216C86803240 @default.
- W4313200216 hasConceptScore W4313200216C92087593 @default.
- W4313200216 hasIssue "0" @default.
- W4313200216 hasLocation W43132002161 @default.
- W4313200216 hasOpenAccess W4313200216 @default.
- W4313200216 hasPrimaryLocation W43132002161 @default.
- W4313200216 hasRelatedWork W1965116784 @default.
- W4313200216 hasRelatedWork W1981508914 @default.
- W4313200216 hasRelatedWork W2038573040 @default.
- W4313200216 hasRelatedWork W2386081253 @default.
- W4313200216 hasRelatedWork W3004173670 @default.
- W4313200216 hasRelatedWork W3205113062 @default.
- W4313200216 hasRelatedWork W4205890851 @default.
- W4313200216 hasRelatedWork W4206755691 @default.
- W4313200216 hasRelatedWork W4230735107 @default.
- W4313200216 hasRelatedWork W4298111954 @default.
- W4313200216 hasVolume "96" @default.
- W4313200216 isParatext "false" @default.
- W4313200216 isRetracted "false" @default.
- W4313200216 workType "article" @default.