Matches in SemOpenAlex for { <https://semopenalex.org/work/W2892448852> ?p ?o ?g. }
- W2892448852 endingPage "206" @default.
- W2892448852 startingPage "191" @default.
- W2892448852 abstract "Hemoglobinopathies are inherited genetic conditions that originate from a lack or malfunction of the adult hemoglobin protein. Thalassemia and other diseases associated with β-globin abnormal amino acid sequences—such as sickle cell disease (SCD) and hemoglobin E (HbE)—are some of the most common hemoglobinopathies. Severe anemia combined with complications that arise in the most affected patients raises the necessity for a cure to restore hemoglobin function. The current routine therapies for these conditions, namely transfusion and iron chelation, have significantly improved the quality of life in patients over the years, but still fail to address the underlying cause of the diseases. A curative option, allogeneic bone marrow (BM) transplantation, is available, but is limited by the availability of suitable donors and graft-versus-host disease (GVHD). Gene therapy offers an alternative approach to cure patients with hemoglobinopathies and aims at the direct recovery of the hemoglobin function via globin gene transfer. In the last two decades, gene transfer tools based on lentiviral vector development have been significantly improved, and proven curative in several animal models for SCD and thalassemia. As a result, clinical trials are in progress and three patients have been successfully treated with this approach. However, there are still frontiers to explore that might improve this approach: the stoichiometry between the transgenic hemoglobin and endogenous hemoglobin with respect to the different globin genetic mutations; donor cell sourcing, such as the use of induced pluripotent stem cells (iPSCs); and the use of safer gene insertion methods to prevent oncogenesis. This review will provide insights into the different lentiviral gene therapy approaches in mouse models and human cells; current and planned clinical trials; hurdles to overcome for clinical trials, such as myeloablation toxicity, insertional oncogenesis, and high vector expression; and future perspectives for gene therapy, including safe harbors, reactivation of fetal hemoglobin, and iPSC technology." @default.
- W2892448852 created "2018-10-05" @default.
- W2892448852 creator A5046499318 @default.
- W2892448852 creator A5069433670 @default.
- W2892448852 creator A5075482645 @default.
- W2892448852 date "2015-01-01" @default.
- W2892448852 modified "2023-09-27" @default.
- W2892448852 title "Gene Therapy for Hemoglobinopathies" @default.
- W2892448852 cites W119649542 @default.
- W2892448852 cites W1554364555 @default.
- W2892448852 cites W1595738627 @default.
- W2892448852 cites W1598569053 @default.
- W2892448852 cites W1697357186 @default.
- W2892448852 cites W1835972708 @default.
- W2892448852 cites W1966991523 @default.
- W2892448852 cites W1968034459 @default.
- W2892448852 cites W1969826189 @default.
- W2892448852 cites W1970831058 @default.
- W2892448852 cites W1973669128 @default.
- W2892448852 cites W1974572528 @default.
- W2892448852 cites W1976626122 @default.
- W2892448852 cites W1980449150 @default.
- W2892448852 cites W1981268254 @default.
- W2892448852 cites W1981481637 @default.
- W2892448852 cites W1982360832 @default.
- W2892448852 cites W1982564326 @default.
- W2892448852 cites W1983173999 @default.
- W2892448852 cites W1983436475 @default.
- W2892448852 cites W1984844550 @default.
- W2892448852 cites W1984858697 @default.
- W2892448852 cites W1986744301 @default.
- W2892448852 cites W1991062799 @default.
- W2892448852 cites W1997136467 @default.
- W2892448852 cites W1997170085 @default.
- W2892448852 cites W2002456771 @default.
- W2892448852 cites W2003103876 @default.
- W2892448852 cites W2005655057 @default.
- W2892448852 cites W2007352439 @default.
- W2892448852 cites W2007665650 @default.
- W2892448852 cites W2008000491 @default.
- W2892448852 cites W2008331547 @default.
- W2892448852 cites W2008685326 @default.
- W2892448852 cites W2008973770 @default.
- W2892448852 cites W2008989169 @default.
- W2892448852 cites W2009043079 @default.
- W2892448852 cites W2009761723 @default.
- W2892448852 cites W2009938167 @default.
- W2892448852 cites W2010031978 @default.
- W2892448852 cites W2010874858 @default.
- W2892448852 cites W2011686157 @default.
- W2892448852 cites W2011695731 @default.
- W2892448852 cites W2015378087 @default.
- W2892448852 cites W2017202140 @default.
- W2892448852 cites W2019294341 @default.
- W2892448852 cites W2020397724 @default.
- W2892448852 cites W2023449596 @default.
- W2892448852 cites W2024235343 @default.
- W2892448852 cites W2024725095 @default.
- W2892448852 cites W2024999114 @default.
- W2892448852 cites W2025275264 @default.
- W2892448852 cites W2027401034 @default.
- W2892448852 cites W2028273079 @default.
- W2892448852 cites W2028369290 @default.
- W2892448852 cites W2029399500 @default.
- W2892448852 cites W2031532553 @default.
- W2892448852 cites W2032487385 @default.
- W2892448852 cites W2032788328 @default.
- W2892448852 cites W2033380734 @default.
- W2892448852 cites W2035121417 @default.
- W2892448852 cites W2037473193 @default.
- W2892448852 cites W2038815755 @default.
- W2892448852 cites W2038935839 @default.
- W2892448852 cites W2042024930 @default.
- W2892448852 cites W2049271350 @default.
- W2892448852 cites W2049577511 @default.
- W2892448852 cites W2049742620 @default.
- W2892448852 cites W2052012202 @default.
- W2892448852 cites W2052150955 @default.
- W2892448852 cites W2052657961 @default.
- W2892448852 cites W2053356302 @default.
- W2892448852 cites W2053576250 @default.
- W2892448852 cites W2053911973 @default.
- W2892448852 cites W2054406966 @default.
- W2892448852 cites W2064820560 @default.
- W2892448852 cites W2066099080 @default.
- W2892448852 cites W2066783828 @default.
- W2892448852 cites W2067032417 @default.
- W2892448852 cites W2072692007 @default.
- W2892448852 cites W2074957003 @default.
- W2892448852 cites W2078752671 @default.
- W2892448852 cites W2080008193 @default.
- W2892448852 cites W2086284653 @default.
- W2892448852 cites W2089305246 @default.
- W2892448852 cites W2092627970 @default.
- W2892448852 cites W2093537780 @default.
- W2892448852 cites W2093750771 @default.
- W2892448852 cites W2094204764 @default.
- W2892448852 cites W2096529455 @default.