197 related articles for article (PubMed ID: 34139105)
1. A systems pharmacology model for gene therapy in sickle cell disease.
Zheng B; Wille L; Peppel K; Hagen D; Matteson A; Ahlers J; Schaff J; Hua F; Yuraszeck T; Cobbina E; Apgar JF; Burke JM; Roberts J; Das R
CPT Pharmacometrics Syst Pharmacol; 2021 Jul; 10(7):696-708. PubMed ID: 34139105
[TBL] [Abstract][Full Text] [Related]
2. Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease.
Park SH; Lee CM; Dever DP; Davis TH; Camarena J; Srifa W; Zhang Y; Paikari A; Chang AK; Porteus MH; Sheehan VA; Bao G
Nucleic Acids Res; 2019 Sep; 47(15):7955-7972. PubMed ID: 31147717
[TBL] [Abstract][Full Text] [Related]
3. Cellular function reinstitution of offspring red blood cells cloned from the sickle cell disease patient blood post CRISPR genome editing.
Wen J; Tao W; Hao S; Zu Y
J Hematol Oncol; 2017 Jun; 10(1):119. PubMed ID: 28610635
[TBL] [Abstract][Full Text] [Related]
4. Mathematical modeling of erythrocyte chimerism informs genetic intervention strategies for sickle cell disease.
Altrock PM; Brendel C; Renella R; Orkin SH; Williams DA; Michor F
Am J Hematol; 2016 Sep; 91(9):931-7. PubMed ID: 27299299
[TBL] [Abstract][Full Text] [Related]
5. Gene replacement therapy for sickle cell disease and other blood disorders.
Townes TM
Hematology Am Soc Hematol Educ Program; 2008; ():193-6. PubMed ID: 19074080
[TBL] [Abstract][Full Text] [Related]
6. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study.
Urbinati F; Campo Fernandez B; Masiuk KE; Poletti V; Hollis RP; Koziol C; Kaufman ML; Brown D; Mavilio F; Kohn DB
Hum Gene Ther; 2018 Oct; 29(10):1153-1166. PubMed ID: 30198339
[TBL] [Abstract][Full Text] [Related]
7. Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
Breda L; Casu C; Gardenghi S; Bianchi N; Cartegni L; Narla M; Yazdanbakhsh K; Musso M; Manwani D; Little J; Gardner LB; Kleinert DA; Prus E; Fibach E; Grady RW; Giardina PJ; Gambari R; Rivella S
PLoS One; 2012; 7(3):e32345. PubMed ID: 22479321
[TBL] [Abstract][Full Text] [Related]
8. Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.
Kanter J; Thompson AA; Pierciey FJ; Hsieh M; Uchida N; Leboulch P; Schmidt M; Bonner M; Guo R; Miller A; Ribeil JA; Davidson D; Asmal M; Walters MC; Tisdale JF
Am J Hematol; 2023 Jan; 98(1):11-22. PubMed ID: 36161320
[TBL] [Abstract][Full Text] [Related]
9. β-Globin sleeping beauty transposon reduces red blood cell sickling in a patient-derived CD34(+)-based in vitro model.
Sjeklocha LM; Wong PY; Belcher JD; Vercellotti GM; Steer CJ
PLoS One; 2013; 8(11):e80403. PubMed ID: 24260386
[TBL] [Abstract][Full Text] [Related]
10. Diverse Approaches to Gene Therapy of Sickle Cell Disease.
White SL; Hart K; Kohn DB
Annu Rev Med; 2023 Jan; 74():473-487. PubMed ID: 36067800
[TBL] [Abstract][Full Text] [Related]
11. A cure for murine sickle cell disease through stable mixed chimerism and tolerance induction after nonmyeloablative conditioning and major histocompatibility complex-mismatched bone marrow transplantation.
Kean LS; Durham MM; Adams AB; Hsu LL; Perry JR; Dillehay D; Pearson TC; Waller EK; Larsen CP; Archer DR
Blood; 2002 Mar; 99(5):1840-9. PubMed ID: 11861303
[TBL] [Abstract][Full Text] [Related]
12. Bone Marrow as a Hematopoietic Stem Cell Source for Gene Therapy in Sickle Cell Disease: Evidence from Rhesus and SCD Patients.
Uchida N; Fujita A; Hsieh MM; Bonifacino AC; Krouse AE; Metzger ME; Donahue RE; Tisdale JF
Hum Gene Ther Clin Dev; 2017 Sep; 28(3):136-144. PubMed ID: 28447889
[TBL] [Abstract][Full Text] [Related]
13. Engraftment of immune-deficient mice with primitive hematopoietic cells from beta-thalassemia and sickle cell anemia patients: implications for evaluating human gene therapy protocols.
Larochelle A; Vormoor J; Lapidot T; Sher G; Furukawa T; Li Q; Shultz LD; Olivieri NF; Stamatoyannopoulos G; Dick JE
Hum Mol Genet; 1995 Feb; 4(2):163-72. PubMed ID: 7757063
[TBL] [Abstract][Full Text] [Related]
14. A novel human gamma-globin gene vector for genetic correction of sickle cell anemia in a humanized sickle mouse model: critical determinants for successful correction.
Perumbeti A; Higashimoto T; Urbinati F; Franco R; Meiselman HJ; Witte D; Malik P
Blood; 2009 Aug; 114(6):1174-85. PubMed ID: 19474450
[TBL] [Abstract][Full Text] [Related]
15. Bone Marrow Transplantation after Nonmyeloablative Treosulfan Conditioning Is Curative in a Murine Model of Sickle Cell Disease.
Devadasan D; Sun CW; Westin ER; Wu LC; Pawlik KM; Townes TM; Goldman FD
Biol Blood Marrow Transplant; 2018 Aug; 24(8):1554-1562. PubMed ID: 29684562
[TBL] [Abstract][Full Text] [Related]
16. Current state of gene therapy in sickle cell disease.
Tang MS; Shan H
Vox Sang; 2024 Jun; 119(6):521-528. PubMed ID: 38487952
[TBL] [Abstract][Full Text] [Related]
17. A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease.
Hart KL; Liu B; Brown D; Campo-Fernandez B; Tam K; Orr K; Hollis RP; Brendel C; Williams DA; Kohn DB
Mol Ther Methods Clin Dev; 2024 Jun; 32(2):101254. PubMed ID: 38745893
[TBL] [Abstract][Full Text] [Related]
18. Chimerism and cure: hematologic and pathologic correction of murine sickle cell disease.
Kean LS; Manci EA; Perry J; Balkan C; Coley S; Holtzclaw D; Adams AB; Larsen CP; Hsu LL; Archer DR
Blood; 2003 Dec; 102(13):4582-93. PubMed ID: 12933586
[TBL] [Abstract][Full Text] [Related]
19. Gene Therapy Approaches to Hemoglobinopathies.
Ferrari G; Cavazzana M; Mavilio F
Hematol Oncol Clin North Am; 2017 Oct; 31(5):835-852. PubMed ID: 28895851
[TBL] [Abstract][Full Text] [Related]
20. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease.
Poletti V; Urbinati F; Charrier S; Corre G; Hollis RP; Campo Fernandez B; Martin S; Rothe M; Schambach A; Kohn DB; Mavilio F
Mol Ther Methods Clin Dev; 2018 Dec; 11():167-179. PubMed ID: 30533448
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]