FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4207036299> ?p ?o ?g. }

• W4207036299 endingPage "80" @default.
• W4207036299 startingPage "71" @default.
• W4207036299 abstract "Patients with Wiskott-Aldrich syndrome (WAS) lacking a human leukocyte antigen-matched donor may benefit from gene therapy through the provision of gene-corrected, autologous hematopoietic stem/progenitor cells. Here, we present comprehensive, long-term follow-up results (median follow-up, 7.6 years) (phase I/II trial no. NCT02333760 ) for eight patients with WAS having undergone phase I/II lentiviral vector-based gene therapy trials (nos. NCT01347346 and NCT01347242 ), with a focus on thrombocytopenia and autoimmunity. Primary outcomes of the long-term study were to establish clinical and biological safety, efficacy and tolerability by evaluating the incidence and type of serious adverse events and clinical status and biological parameters including lentiviral genomic integration sites in different cell subpopulations from 3 years to 15 years after gene therapy. Secondary outcomes included monitoring the need for additional treatment and T cell repertoire diversity. An interim analysis shows that the study meets the primary outcome criteria tested given that the gene-corrected cells engrafted stably, and no serious treatment-associated adverse events occurred. Overall, severe infections and eczema resolved. Autoimmune disorders and bleeding episodes were significantly less frequent, despite only partial correction of the platelet compartment. The results suggest that lentiviral gene therapy provides sustained clinical benefits for patients with WAS." @default.
• W4207036299 created "2022-01-26" @default.
• W4207036299 creator A5000888675 @default.
• W4207036299 creator A5001270472 @default.
• W4207036299 creator A5002366090 @default.
• W4207036299 creator A5003228879 @default.
• W4207036299 creator A5003912397 @default.
• W4207036299 creator A5004972535 @default.
• W4207036299 creator A5005104278 @default.
• W4207036299 creator A5007387587 @default.
• W4207036299 creator A5008770089 @default.
• W4207036299 creator A5010488274 @default.
• W4207036299 creator A5016101702 @default.
• W4207036299 creator A5017005000 @default.
• W4207036299 creator A5018924006 @default.
• W4207036299 creator A5022932915 @default.
• W4207036299 creator A5023954428 @default.
• W4207036299 creator A5028292912 @default.
• W4207036299 creator A5029004287 @default.
• W4207036299 creator A5032103104 @default.
• W4207036299 creator A5033775906 @default.
• W4207036299 creator A5041625192 @default.
• W4207036299 creator A5042010611 @default.
• W4207036299 creator A5043452031 @default.
• W4207036299 creator A5043754495 @default.
• W4207036299 creator A5053217321 @default.
• W4207036299 creator A5053965677 @default.
• W4207036299 creator A5057274300 @default.
• W4207036299 creator A5061478893 @default.
• W4207036299 creator A5063790935 @default.
• W4207036299 creator A5064789849 @default.
• W4207036299 creator A5072081600 @default.
• W4207036299 creator A5072838648 @default.
• W4207036299 creator A5073545087 @default.
• W4207036299 creator A5074760478 @default.
• W4207036299 creator A5075374054 @default.
• W4207036299 creator A5075939022 @default.
• W4207036299 creator A5078600514 @default.
• W4207036299 creator A5080648054 @default.
• W4207036299 creator A5081606844 @default.
• W4207036299 creator A5087348143 @default.
• W4207036299 creator A5091417698 @default.
• W4207036299 date "2022-01-01" @default.
• W4207036299 modified "2023-10-18" @default.
• W4207036299 title "Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome" @default.
• W4207036299 cites W1590348274 @default.
• W4207036299 cites W1780006609 @default.
• W4207036299 cites W1981070200 @default.
• W4207036299 cites W1981881077 @default.
• W4207036299 cites W1984241869 @default.
• W4207036299 cites W1984284869 @default.
• W4207036299 cites W1985160996 @default.
• W4207036299 cites W1990338101 @default.
• W4207036299 cites W1993483086 @default.
• W4207036299 cites W2014304617 @default.
• W4207036299 cites W2034552117 @default.
• W4207036299 cites W2034609134 @default.
• W4207036299 cites W2048538932 @default.
• W4207036299 cites W2050419057 @default.
• W4207036299 cites W2070638961 @default.
• W4207036299 cites W2074477988 @default.
• W4207036299 cites W2087968292 @default.
• W4207036299 cites W2089457797 @default.
• W4207036299 cites W2092668505 @default.
• W4207036299 cites W2095089544 @default.
• W4207036299 cites W2103771488 @default.
• W4207036299 cites W2116821263 @default.
• W4207036299 cites W2118340386 @default.
• W4207036299 cites W2130174662 @default.
• W4207036299 cites W2142159826 @default.
• W4207036299 cites W2153812915 @default.
• W4207036299 cites W2169127974 @default.
• W4207036299 cites W2171724615 @default.
• W4207036299 cites W2288675092 @default.
• W4207036299 cites W2562817659 @default.
• W4207036299 cites W2565961003 @default.
• W4207036299 cites W2566544067 @default.
• W4207036299 cites W2593244680 @default.
• W4207036299 cites W2735267401 @default.
• W4207036299 cites W2766769159 @default.
• W4207036299 cites W2784053360 @default.
• W4207036299 cites W2784975031 @default.
• W4207036299 cites W2785911985 @default.
• W4207036299 cites W2789297879 @default.
• W4207036299 cites W2883107064 @default.
• W4207036299 cites W2883568907 @default.
• W4207036299 cites W2891016653 @default.
• W4207036299 cites W2921224169 @default.
• W4207036299 cites W2924007987 @default.
• W4207036299 cites W2938307622 @default.
• W4207036299 cites W2954189331 @default.
• W4207036299 cites W3006279252 @default.
• W4207036299 cites W3014992883 @default.
• W4207036299 cites W3087180166 @default.
• W4207036299 cites W3169349 @default.
• W4207036299 cites W86341414 @default.
• W4207036299 doi "https://doi.org/10.1038/s41591-021-01641-x" @default.
• W4207036299 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/35075289" @default.

• next