Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Scott Witting"'
Autor:
Omar Niss, Monika Asnani, Alisa Dong, Punam Malik, Archana Shrestha, Carolyn Lutzko, Charles T. Quinn, Paritha Arumugam, Sydney Felker, Courtney R Little, Scott Witting, Chris Lo, Jennifer Knight-Madden, Michael Grimley
Publikováno v:
Blood. 138:3970-3970
Introduction: Sickle cell disease (SCD) is a genetic red blood cell (RBC) disorder that causes chronic hemolytic anemia, progressive organ damage, and life-threatening acute complications such as painful vaso-occlusive crises. Allogeneic hematopoieti
Autor:
Charles T. Quinn, Monika Asnani, Punam Malik, Archana Shrestha, Chris Lo, Omar Niss, Joseph W. McIntosh, Paritha Arumugam, Carolyn Lutzko, Courtney R Little, Scott Witting, Jennifer Knight-Madden, Michael Grimley, Sydney Felker
Publikováno v:
Blood. 136:20-21
Introduction: ARU-1801 is a gene therapy consisting of autologous CD34+ hematopoietic stem cells and progenitors (HSCPs) transduced with a lentiviral vector (LV) encoding a modified γ-globinG16D gene. Preclinical studies in SCD mice have shown that
Publikováno v:
Methods in Molecular Biology ISBN: 9781493990641
Lentiviral vectors have rapidly become a favorite tool for research and clinical gene transfer applications which seek to permanently introduce alterations in the genome. This status can be attributed primarily to their ability to transduce dividing
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::af0eb9098c01765fc75e7fdd0eb21aa8
https://doi.org/10.1007/978-1-4939-9065-8_8
https://doi.org/10.1007/978-1-4939-9065-8_8
Autor:
Stella M. Davies, Archana Shreshta, Johannes C.M. Van der Loo, Jennifer Knight-Madden, Theodosia A. Kalfa, Frederic D. Bushman, Monika Asnani, Michael Grimley, Punam Malik, Omar Niss, Elke Grassman, Carolyn Lutzko, Lilith Reeves, Parinda A. Mehta, Amy Shova, Catherine E. Terrell, Sydney Felker, Loberg Anastacia, Charles T. Quinn, Karen Kalinyak, Scott Witting, Little Courtney, Sharat Chandra, Devin Pillis, Diana Nordling
Publikováno v:
Blood. 132:1021-1021
Background: Genetic transfer of an anti-sickling β87-globin lentiviral vector (LV) into hematopoietic stem cells (HSC) followed by myeloablative transplant has cured one child with sickle cell anemia (SCA) (NEJM 2017), although it was not successful
Publikováno v:
Molecular Therapy. 24:S181-S182
Gene delivery using viral vectors is a powerful tool which can be used to correct genetic diseases and treat acquired illnesses. HIV-based lentiviral vectors (LVs) are advantageous in gene therapy applications for due to their genomic integration and
Publikováno v:
Molecular Therapy. 24:S279
Current regulatory guidelines require RCL testing of lentiviral vector preparations used in human clinical trials. Cells transduced ex vivo and cultured four days or more must also be tested before administering to the patient. Attenuated HIV (R8.71)