171 related articles for article (PubMed ID: 33789438)
1. Long-Term Follow-Up of Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Bougnères P; Hacein-Bey-Abina S; Labik I; Adamsbaum C; Castaignède C; Bellesme C; Schmidt M
Hum Gene Ther; 2021 Oct; 32(19-20):1260-1269. PubMed ID: 33789438
[TBL] [Abstract][Full Text] [Related]
2. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F; Duncan C; Musolino PL; Orchard PJ; De Oliveira S; Thrasher AJ; Armant M; Dansereau C; Lund TC; Miller WP; Raymond GV; Sankar R; Shah AJ; Sevin C; Gaspar HB; Gissen P; Amartino H; Bratkovic D; Smith NJC; Paker AM; Shamir E; O'Meara T; Davidson D; Aubourg P; Williams DA
N Engl J Med; 2017 Oct; 377(17):1630-1638. PubMed ID: 28976817
[TBL] [Abstract][Full Text] [Related]
3. Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy.
Cartier N; Hacein-Bey-Abina S; Bartholomae CC; Bougnères P; Schmidt M; Kalle CV; Fischer A; Cavazzana-Calvo M; Aubourg P
Methods Enzymol; 2012; 507():187-98. PubMed ID: 22365775
[TBL] [Abstract][Full Text] [Related]
4. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
Cartier N; Hacein-Bey-Abina S; Bartholomae CC; Veres G; Schmidt M; Kutschera I; Vidaud M; Abel U; Dal-Cortivo L; Caccavelli L; Mahlaoui N; Kiermer V; Mittelstaedt D; Bellesme C; Lahlou N; Lefrère F; Blanche S; Audit M; Payen E; Leboulch P; l'Homme B; Bougnères P; Von Kalle C; Fischer A; Cavazzana-Calvo M; Aubourg P
Science; 2009 Nov; 326(5954):818-23. PubMed ID: 19892975
[TBL] [Abstract][Full Text] [Related]
5. [Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector].
Cartier N; Hacein-Bey-Abina S; Von Kalle C; Bougnères P; Fischer A; Cavazzana-Calvo M; Aubourg P
Bull Acad Natl Med; 2010 Feb; 194(2):255-64; discussion 264-8. PubMed ID: 21166117
[TBL] [Abstract][Full Text] [Related]
6. Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy.
Cartier N; Aubourg P
Brain Pathol; 2010 Jul; 20(4):857-62. PubMed ID: 20626747
[TBL] [Abstract][Full Text] [Related]
7. Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice.
Benhamida S; Pflumio F; Dubart-Kupperschmitt A; Zhao-Emonet JC; Cavazzana-Calvo M; Rocchiccioli F; Fichelson S; Aubourg P; Charneau P; Cartier N
Mol Ther; 2003 Mar; 7(3):317-24. PubMed ID: 12668127
[TBL] [Abstract][Full Text] [Related]
8. Potential Risks to Stable Long-term Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Children With Cerebral X-linked Adrenoleukodystrophy.
Kühl JS; Kupper J; Baqué H; Ebell W; Gärtner J; Korenke C; Spors B; Steffen IG; Strauss G; Voigt S; Weschke B; Weddige A; Köhler W; Steinfeld R
JAMA Netw Open; 2018 Jul; 1(3):e180769. PubMed ID: 30646031
[TBL] [Abstract][Full Text] [Related]
9. Neurocognitive Trajectory of Boys Who Received a Hematopoietic Stem Cell Transplant at an Early Stage of Childhood Cerebral Adrenoleukodystrophy.
Pierpont EI; Eisengart JB; Shanley R; Nascene D; Raymond GV; Shapiro EG; Ziegler RS; Orchard PJ; Miller WP
JAMA Neurol; 2017 Jun; 74(6):710-717. PubMed ID: 28418523
[TBL] [Abstract][Full Text] [Related]
10. Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy.
Orchard PJ; Nascene DR; Miller WP; Gupta A; Kenney-Jung D; Lund TC
Blood; 2019 Mar; 133(12):1378-1381. PubMed ID: 30635285
[TBL] [Abstract][Full Text] [Related]
11. Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy.
Gupta AO; Raymond G; Pierpont EI; Kemp S; McIvor RS; Rayannavar A; Miller B; Lund TC; Orchard PJ
Expert Opin Biol Ther; 2022 Sep; 22(9):1151-1162. PubMed ID: 36107226
[TBL] [Abstract][Full Text] [Related]
12. Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy.
Lauer A; Speroni SL; Choi M; Da X; Duncan C; McCarthy S; Krishnan V; Lusk CA; Rohde D; Hansen MB; Kalpathy-Cramer J; Loes DJ; Caruso PA; Williams DA; Mouridsen K; Emblem KE; Eichler FS; Musolino PL
Nat Commun; 2023 Apr; 14(1):1900. PubMed ID: 37019892
[TBL] [Abstract][Full Text] [Related]
13. Long-term outcomes of allogeneic haematopoietic stem cell transplantation for adult cerebral X-linked adrenoleukodystrophy.
Kühl JS; Suarez F; Gillett GT; Hemmati PG; Snowden JA; Stadler M; Vuong GL; Aubourg P; Köhler W; Arnold R
Brain; 2017 Apr; 140(4):953-966. PubMed ID: 28375456
[TBL] [Abstract][Full Text] [Related]
14. Gene therapy strategies for X-linked adrenoleukodystrophy.
Cartier N
Curr Opin Mol Ther; 2001 Aug; 3(4):357-61. PubMed ID: 11525559
[TBL] [Abstract][Full Text] [Related]
15. The Changing Face of Adrenoleukodystrophy.
Zhu J; Eichler F; Biffi A; Duncan CN; Williams DA; Majzoub JA
Endocr Rev; 2020 Aug; 41(4):577-93. PubMed ID: 32364223
[TBL] [Abstract][Full Text] [Related]
16. Predictive factors for vision loss after hematopoietic cell transplant for X-linked adrenoleukodystrophy.
Gess A; Christiansen SP; Pond D; Peters C
J AAPOS; 2008 Jun; 12(3):273-6. PubMed ID: 18359652
[TBL] [Abstract][Full Text] [Related]
17. Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy.
Pierpont EI; Nascene DR; Shanley R; Kenney-Jung DL; Ziegler RS; Miller WP; Gupta AO; Lund TC; Orchard PJ; Eisengart JB
Neurology; 2020 Aug; 95(5):e591-e600. PubMed ID: 32616675
[TBL] [Abstract][Full Text] [Related]
18. [Hematopoietic cell transplantation in X-linked adrenoleukodystrophy].
Kato S
Brain Nerve; 2007 Apr; 59(4):339-46. PubMed ID: 17447520
[TBL] [Abstract][Full Text] [Related]
19. Retroviral transfer and long-term expression of the adrenoleukodystrophy gene in human CD34+ cells.
Doerflinger N; Miclea JM; Lopez J; Chomienne C; Bougnères P; Aubourg P; Cartier N
Hum Gene Ther; 1998 May; 9(7):1025-36. PubMed ID: 9607414
[TBL] [Abstract][Full Text] [Related]
20. Co-expression of mutated and normal adrenoleukodystrophy protein reduces protein function: implications for gene therapy of X-linked adrenoleukodystrophy.
Unterrainer G; Molzer B; Forss-Petter S; Berger J
Hum Mol Genet; 2000 Nov; 9(18):2609-16. PubMed ID: 11063720
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
