FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W2597658107> ?p ?o ?g. }

• W2597658107 endingPage "1699" @default.
• W2597658107 startingPage "1689" @default.
• W2597658107 abstract "Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and allogeneic HSCT that has shown clinical benefit for adenosine deaminase-deficient (ADA-deficient) SCID when combined with reduced intensity conditioning (RIC) and ERT cessation. Clinical safety and therapeutic efficacy were evaluated in a phase II study.Ten subjects with confirmed ADA-deficient SCID and no available matched sibling or family donor were enrolled between 2009 and 2012 and received transplantation with autologous hematopoietic CD34+ cells that were modified with the human ADA cDNA (MND-ADA) γ-retroviral vector after conditioning with busulfan (90 mg/m2) and ERT cessation. Subjects were followed from 33 to 84 months at the time of data analysis. Safety of the procedure was assessed by recording the number of adverse events. Efficacy was assessed by measuring engraftment of gene-modified hematopoietic stem/progenitor cells, ADA gene expression, and immune reconstitution.With the exception of the oldest subject (15 years old at enrollment), all subjects remained off ERT with normalized peripheral blood mononuclear cell (PBMC) ADA activity, improved lymphocyte numbers, and normal proliferative responses to mitogens. Three of nine subjects were able to discontinue intravenous immunoglobulin replacement therapy. The MND-ADA vector was persistently detected in PBMCs (vector copy number [VCN] = 0.1-2.6) and granulocytes (VCN = 0.01-0.3) through the most recent visits at the time of this writing. No patient has developed a leukoproliferative disorder or other vector-related clinical complication since transplant.These results demonstrate clinical therapeutic efficacy from gene therapy for ADA-deficient SCID, with an excellent clinical safety profile.ClinicalTrials.gov NCT00794508.Food and Drug Administration Office of Orphan Product Development award, RO1 FD003005; NHLBI awards, PO1 HL73104 and Z01 HG000122; UCLA Clinical and Translational Science Institute awards, UL1RR033176 and UL1TR000124." @default.
• W2597658107 created "2017-04-07" @default.
• W2597658107 creator A5000575140 @default.
• W2597658107 creator A5002793183 @default.
• W2597658107 creator A5005379690 @default.
• W2597658107 creator A5008643826 @default.
• W2597658107 creator A5009145909 @default.
• W2597658107 creator A5010407904 @default.
• W2597658107 creator A5011722048 @default.
• W2597658107 creator A5014943337 @default.
• W2597658107 creator A5016007010 @default.
• W2597658107 creator A5016164107 @default.
• W2597658107 creator A5021290022 @default.
• W2597658107 creator A5026726927 @default.
• W2597658107 creator A5031526764 @default.
• W2597658107 creator A5031879484 @default.
• W2597658107 creator A5032720690 @default.
• W2597658107 creator A5033477524 @default.
• W2597658107 creator A5035146568 @default.
• W2597658107 creator A5039832836 @default.
• W2597658107 creator A5045989873 @default.
• W2597658107 creator A5048397569 @default.
• W2597658107 creator A5049748479 @default.
• W2597658107 creator A5049810916 @default.
• W2597658107 creator A5050028162 @default.
• W2597658107 creator A5050971129 @default.
• W2597658107 creator A5052887789 @default.
• W2597658107 creator A5054509454 @default.
• W2597658107 creator A5059511446 @default.
• W2597658107 creator A5061818157 @default.
• W2597658107 creator A5061879284 @default.
• W2597658107 creator A5062385004 @default.
• W2597658107 creator A5062526829 @default.
• W2597658107 creator A5063879916 @default.
• W2597658107 creator A5071286209 @default.
• W2597658107 creator A5074244843 @default.
• W2597658107 creator A5074908349 @default.
• W2597658107 creator A5076401965 @default.
• W2597658107 creator A5077876886 @default.
• W2597658107 creator A5080441833 @default.
• W2597658107 creator A5080523462 @default.
• W2597658107 creator A5083017812 @default.
• W2597658107 creator A5084179048 @default.
• W2597658107 creator A5087437800 @default.
• W2597658107 creator A5088091880 @default.
• W2597658107 creator A5091468377 @default.
• W2597658107 date "2017-03-27" @default.
• W2597658107 modified "2023-10-01" @default.
• W2597658107 title "Clinical efficacy of gene-modified stem cells in adenosine deaminase–deficient immunodeficiency" @default.
• W2597658107 cites W1494122660 @default.
• W2597658107 cites W1535428241 @default.
• W2597658107 cites W1548074080 @default.
• W2597658107 cites W1666496871 @default.
• W2597658107 cites W1686812758 @default.
• W2597658107 cites W1856430332 @default.
• W2597658107 cites W1863374972 @default.
• W2597658107 cites W1893260165 @default.
• W2597658107 cites W1965121914 @default.
• W2597658107 cites W1966530560 @default.
• W2597658107 cites W1977486219 @default.
• W2597658107 cites W1978660935 @default.
• W2597658107 cites W2002333878 @default.
• W2597658107 cites W2017608059 @default.
• W2597658107 cites W2024689485 @default.
• W2597658107 cites W2032678451 @default.
• W2597658107 cites W2037256495 @default.
• W2597658107 cites W2039333435 @default.
• W2597658107 cites W2040062791 @default.
• W2597658107 cites W2054619185 @default.
• W2597658107 cites W2056318828 @default.
• W2597658107 cites W2057879405 @default.
• W2597658107 cites W2063909696 @default.
• W2597658107 cites W2066566403 @default.
• W2597658107 cites W2091872525 @default.
• W2597658107 cites W2107046588 @default.
• W2597658107 cites W2107278930 @default.
• W2597658107 cites W2121444412 @default.
• W2597658107 cites W2130174662 @default.
• W2597658107 cites W2137440275 @default.
• W2597658107 cites W2323613689 @default.
• W2597658107 cites W2344579973 @default.
• W2597658107 cites W2510258949 @default.
• W2597658107 cites W3111113094 @default.
• W2597658107 doi "https://doi.org/10.1172/jci90367" @default.
• W2597658107 hasPubMedCentralId "https://www.ncbi.nlm.nih.gov/pmc/articles/5409097" @default.
• W2597658107 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/28346229" @default.
• W2597658107 hasPublicationYear "2017" @default.
• W2597658107 type Work @default.
• W2597658107 sameAs 2597658107 @default.
• W2597658107 citedByCount "69" @default.
• W2597658107 countsByYear W25976581072017 @default.
• W2597658107 countsByYear W25976581072018 @default.
• W2597658107 countsByYear W25976581072019 @default.
• W2597658107 countsByYear W25976581072020 @default.
• W2597658107 countsByYear W25976581072021 @default.
• W2597658107 countsByYear W25976581072022 @default.
• W2597658107 countsByYear W25976581072023 @default.
• W2597658107 crossrefType "journal-article" @default.

• next