Matches in SemOpenAlex for { <https://semopenalex.org/work/W2017101043> ?p ?o ?g. }
Showing items 1 to 95 of
95
with 100 items per page.
- W2017101043 endingPage "99" @default.
- W2017101043 startingPage "95" @default.
- W2017101043 abstract "Over the 2 years covered here, there has been one clinical study in which a normal alpha-1 antitrypsin (AAT) gene was delivered to the nasal epithelium of AAT-deficient subjects using plasmid-liposome complexes; a second study using an adeno-associated vector should begin soon. Although progress in clinical studies has been slow, advances in both viral and nonviral vector designs show considerable promise. Strategies that combine liposome technology with imaginative vector design may permit long-term expression of a normal transgene that is sufficient to achieve therapeutic serum AAT concentrations. While reproducing the normal physiology by targeting normal AAT gene expression to the liver is logical, local expression in lung cells may be less demanding of the technology and offers therapeutic benefits that are produced neither by AAT protein therapy nor by AAT gene therapy targeted to the liver. Developing technologies may permit direct correction of the mutant AAT gene using innovative approaches to in vivo gene repair." @default.
- W2017101043 created "2016-06-24" @default.
- W2017101043 creator A5061640269 @default.
- W2017101043 creator A5079599264 @default.
- W2017101043 date "2003-01-01" @default.
- W2017101043 modified "2023-09-25" @default.
- W2017101043 title "Gene Therapy Progress and Prospects: Alpha-1 antitrypsin" @default.
- W2017101043 cites W1984183802 @default.
- W2017101043 cites W1999707071 @default.
- W2017101043 cites W2008776042 @default.
- W2017101043 cites W2050821526 @default.
- W2017101043 cites W2051625599 @default.
- W2017101043 cites W2090495408 @default.
- W2017101043 cites W2110464736 @default.
- W2017101043 cites W2156463705 @default.
- W2017101043 cites W2176002001 @default.
- W2017101043 cites W2331299994 @default.
- W2017101043 doi "https://doi.org/10.1038/sj.gt.3301947" @default.
- W2017101043 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/12571637" @default.
- W2017101043 hasPublicationYear "2003" @default.
- W2017101043 type Work @default.
- W2017101043 sameAs 2017101043 @default.
- W2017101043 citedByCount "60" @default.
- W2017101043 countsByYear W20171010432012 @default.
- W2017101043 countsByYear W20171010432013 @default.
- W2017101043 countsByYear W20171010432014 @default.
- W2017101043 countsByYear W20171010432016 @default.
- W2017101043 countsByYear W20171010432020 @default.
- W2017101043 countsByYear W20171010432021 @default.
- W2017101043 crossrefType "journal-article" @default.
- W2017101043 hasAuthorship W2017101043A5061640269 @default.
- W2017101043 hasAuthorship W2017101043A5079599264 @default.
- W2017101043 hasBestOaLocation W20171010431 @default.
- W2017101043 hasConcept C102230213 @default.
- W2017101043 hasConcept C104317684 @default.
- W2017101043 hasConcept C111599444 @default.
- W2017101043 hasConcept C111829913 @default.
- W2017101043 hasConcept C135983454 @default.
- W2017101043 hasConcept C143065580 @default.
- W2017101043 hasConcept C150194340 @default.
- W2017101043 hasConcept C159110408 @default.
- W2017101043 hasConcept C203014093 @default.
- W2017101043 hasConcept C2775944032 @default.
- W2017101043 hasConcept C32470452 @default.
- W2017101043 hasConcept C40767141 @default.
- W2017101043 hasConcept C49453240 @default.
- W2017101043 hasConcept C502942594 @default.
- W2017101043 hasConcept C54355233 @default.
- W2017101043 hasConcept C60644358 @default.
- W2017101043 hasConcept C64943373 @default.
- W2017101043 hasConcept C71924100 @default.
- W2017101043 hasConcept C86803240 @default.
- W2017101043 hasConcept C92087593 @default.
- W2017101043 hasConceptScore W2017101043C102230213 @default.
- W2017101043 hasConceptScore W2017101043C104317684 @default.
- W2017101043 hasConceptScore W2017101043C111599444 @default.
- W2017101043 hasConceptScore W2017101043C111829913 @default.
- W2017101043 hasConceptScore W2017101043C135983454 @default.
- W2017101043 hasConceptScore W2017101043C143065580 @default.
- W2017101043 hasConceptScore W2017101043C150194340 @default.
- W2017101043 hasConceptScore W2017101043C159110408 @default.
- W2017101043 hasConceptScore W2017101043C203014093 @default.
- W2017101043 hasConceptScore W2017101043C2775944032 @default.
- W2017101043 hasConceptScore W2017101043C32470452 @default.
- W2017101043 hasConceptScore W2017101043C40767141 @default.
- W2017101043 hasConceptScore W2017101043C49453240 @default.
- W2017101043 hasConceptScore W2017101043C502942594 @default.
- W2017101043 hasConceptScore W2017101043C54355233 @default.
- W2017101043 hasConceptScore W2017101043C60644358 @default.
- W2017101043 hasConceptScore W2017101043C64943373 @default.
- W2017101043 hasConceptScore W2017101043C71924100 @default.
- W2017101043 hasConceptScore W2017101043C86803240 @default.
- W2017101043 hasConceptScore W2017101043C92087593 @default.
- W2017101043 hasIssue "2" @default.
- W2017101043 hasLocation W20171010431 @default.
- W2017101043 hasLocation W20171010432 @default.
- W2017101043 hasOpenAccess W2017101043 @default.
- W2017101043 hasPrimaryLocation W20171010431 @default.
- W2017101043 hasRelatedWork W2006519535 @default.
- W2017101043 hasRelatedWork W2008118270 @default.
- W2017101043 hasRelatedWork W2017101043 @default.
- W2017101043 hasRelatedWork W2031465191 @default.
- W2017101043 hasRelatedWork W2062787494 @default.
- W2017101043 hasRelatedWork W2236987864 @default.
- W2017101043 hasRelatedWork W2397865238 @default.
- W2017101043 hasRelatedWork W289467534 @default.
- W2017101043 hasRelatedWork W3204636945 @default.
- W2017101043 hasRelatedWork W4250867061 @default.
- W2017101043 hasVolume "10" @default.
- W2017101043 isParatext "false" @default.
- W2017101043 isRetracted "false" @default.
- W2017101043 magId "2017101043" @default.
- W2017101043 workType "article" @default.