Detalhe da pesquisa
1.
Reactivation of developmentally silenced globin genes by forced chromatin looping.
Cell
; 158(4): 849-860, 2014 Aug 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-25126789
2.
Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.
Mol Ther
; 29(4): 1625-1638, 2021 04 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-33515514
3.
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 ß-thalassemia patient cells restoring HbA production and chain rebalance.
Haematologica
; 106(5): 1433-1442, 2019 May 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-32439726
4.
Development and characterization of cellular biosensors for HTS of erythroid differentiation inducers targeting the transcriptional activity of γ-globin and ß-globin gene promoters.
Anal Bioanal Chem
; 411(29): 7669-7680, 2019 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-31273412
5.
Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers.
Blood
; 128(8): 1139-43, 2016 08 25.
Artigo
em Inglês
| MEDLINE | ID: mdl-27405777
6.
A validated cellular biobank for ß-thalassemia.
J Transl Med
; 14: 255, 2016 09 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-27590532
7.
Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of ß-thalassemia.
Blood
; 134(6): 568-572, 2019 08 08.
Artigo
em Inglês
| MEDLINE | ID: mdl-31151988
8.
Inclusion of a short hairpin RNA targeting BCL11A into a ß-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin.
Haematologica
; 106(10): 2740-2745, 2021 10 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-34047176
9.
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations.
bioRxiv
; 2024 Mar 14.
Artigo
em Inglês
| MEDLINE | ID: mdl-38559013
10.
Elevated CDKN1A (P21) mediates ß-thalassemia erythroid apoptosis, but its loss does not improve ß-thalassemic erythropoiesis.
Blood Adv
; 7(22): 6873-6885, 2023 11 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37672319
11.
In vivo hematopoietic stem cell modification by mRNA delivery.
Science
; 381(6656): 436-443, 2023 07 28.
Artigo
em Inglês
| MEDLINE | ID: mdl-37499029
12.
A combined approach for ß-thalassemia based on gene therapy-mediated adult hemoglobin (HbA) production and fetal hemoglobin (HbF) induction.
Ann Hematol
; 91(8): 1201-13, 2012 Aug.
Artigo
em Inglês
| MEDLINE | ID: mdl-22460946
13.
A probable case of holoprosencephaly with cyclopia in a full-term fetus from a modern skeletal collection.
Int J Paleopathol
; 33: 25-29, 2021 06.
Artigo
em Inglês
| MEDLINE | ID: mdl-33640560
14.
Advances in the identification of deciduous molar tooth germs.
Leg Med (Tokyo)
; 48: 101801, 2021 Feb.
Artigo
em Inglês
| MEDLINE | ID: mdl-33268276
15.
Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.
Blood
; 112(3): 875-85, 2008 Aug 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-18480424
16.
Production of beta-globin and adult hemoglobin following G418 treatment of erythroid precursor cells from homozygous beta(0)39 thalassemia patients.
Am J Hematol
; 84(11): 720-8, 2009 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-19810011
17.
Development of K562 cell clones expressing beta-globin mRNA carrying the beta039 thalassaemia mutation for the screening of correctors of stop-codon mutations.
Biotechnol Appl Biochem
; 54(1): 41-52, 2009 Jul 09.
Artigo
em Inglês
| MEDLINE | ID: mdl-19216718
18.
Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges.
Mol Diagn Ther
; 23(2): 173-186, 2019 04.
Artigo
em Inglês
| MEDLINE | ID: mdl-30701409
19.
Curing Hemoglobinopathies: Challenges and Advances of Conventional and New Gene Therapy Approaches.
Mediterr J Hematol Infect Dis
; 11(1): e2019067, 2019.
Artigo
em Inglês
| MEDLINE | ID: mdl-31700592
20.
[When ubiquitination intertwines with cutaneous melanoma: a mechanism to explore]. / Quand l'ubiquitination se mêle du mélanome cutané - Un mécanisme à explorer.
Med Sci (Paris)
; 40(3): 308-310, 2024 Mar.
Artigo
em Francês
| MEDLINE | ID: mdl-38520112