sequences encoding aav capsid variants Search Results


aav vectors with different capsids encoding the fix gene or its padua fix variant  (uniQure Inc)
90
uniQure Inc aav vectors with different capsids encoding the fix gene or its padua fix variant
Clinical trials of gene therapy products for liver monogenic disorders. Intraportal and intrahepatic routes of administration relate to injection on the portal vein and the hepatic artery respectively. FVIII: factor VIII; FIX: <t> factor IX; </t> HD-adenovirus: helper-dependent adenovirus; LDL: low density lipoprotein; MoML: Moloney murine leukaemia virus; NA: not applicable; OTC: ornithine transcarbamylase; PBGD: porphobilinogen deaminase. Information sources: clinicaltrial.gov (accessed 06/01/2017) and company websites. If the date of the start of the trial was not available in clinicaltrials.gov website, the date of publication of the results is mentioned. The list of trials announced for 2017 is indicative and does not pretend to be exhaustive
Aav Vectors With Different Capsids Encoding The Fix Gene Or Its Padua Fix Variant, supplied by uniQure Inc, used in various techniques. Bioz Stars score: 90/100, based on 1 PubMed citations. ZERO BIAS - scores, article reviews, protocol conditions and more
https://www.bioz.com/product/sequences+encoding+aav+capsid+variants/pmc05500673-208-13-42?v=uniQure+Inc
Average 90 stars, based on 1 article reviews
aav vectors with different capsids encoding the fix gene or its padua fix variant - by Bioz Stars, 2026-08
90/100 stars
  Buy from Supplier

Image Search Results


Clinical trials of gene therapy products for liver monogenic disorders. Intraportal and intrahepatic routes of administration relate to injection on the portal vein and the hepatic artery respectively. FVIII: factor VIII; FIX: factor IX; HD-adenovirus: helper-dependent adenovirus; LDL: low density lipoprotein; MoML: Moloney murine leukaemia virus; NA: not applicable; OTC: ornithine transcarbamylase; PBGD: porphobilinogen deaminase. Information sources: clinicaltrial.gov (accessed 06/01/2017) and company websites. If the date of the start of the trial was not available in clinicaltrials.gov website, the date of publication of the results is mentioned. The list of trials announced for 2017 is indicative and does not pretend to be exhaustive

Journal: Journal of Inherited Metabolic Disease

Article Title: Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

doi: 10.1007/s10545-017-0053-3

Figure Lengend Snippet: Clinical trials of gene therapy products for liver monogenic disorders. Intraportal and intrahepatic routes of administration relate to injection on the portal vein and the hepatic artery respectively. FVIII: factor VIII; FIX: factor IX; HD-adenovirus: helper-dependent adenovirus; LDL: low density lipoprotein; MoML: Moloney murine leukaemia virus; NA: not applicable; OTC: ornithine transcarbamylase; PBGD: porphobilinogen deaminase. Information sources: clinicaltrial.gov (accessed 06/01/2017) and company websites. If the date of the start of the trial was not available in clinicaltrials.gov website, the date of publication of the results is mentioned. The list of trials announced for 2017 is indicative and does not pretend to be exhaustive

Article Snippet: After a single intravenous injection of AAV vectors with different capsids encoding the FIX gene or its Padua FIX variant, which contains a gain-of-function mutation, reported stabilised plasma FIX levels have ranged from 3 to 8% in the AMT-060 trial sponsored by Uniqure (Miesbach et al ) and the DTX-101 sponsored by Dimension Therapeutics ( http://dimensiontx.com ) to 20–44% in the high-dose cohort of the BAX 335 trial sponsored by Shire (Monahan et al ) and in the SPK-9001 trial sponsored by Spark Therapeutics/Pfizer (George et al ).

Techniques: Injection, Plasmid Preparation, Ex Vivo, Mutagenesis