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| Result Number | Material Type | Add to My Shelf Action | Record Details and Options |
|---|---|---|---|
| 1 |
Material Type: Artigo
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β-ThalassemiasTaher, Ali T ; Musallam, Khaled M ; Cappellini, M. Domenica Longo, Dan L ; Longo, Dan L.The New England journal of medicine, 2021-02, Vol.384 (8), p.727-743 [Periódico revisado por pares]United States: Massachusetts Medical SocietyTexto completo disponível |
| 2 |
Material Type: Artigo
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Highly efficient therapeutic gene editing of human hematopoietic stem cellsWu, Yuxuan ; Zeng, Jing ; Roscoe, Benjamin P ; Liu, Pengpeng ; Yao, Qiuming ; Lazzarotto, Cicera R ; Clement, Kendell ; Cole, Mitchel A ; Luk, Kevin ; Baricordi, Cristina ; Shen, Anne H ; Ren, Chunyan ; Esrick, Erica B ; Manis, John P ; Dorfman, David M ; Williams, David A ; Biffi, Alessandra ; Brugnara, Carlo ; Biasco, Luca ; Brendel, Christian ; Pinello, Luca ; Tsai, Shengdar Q ; Wolfe, Scot A ; Bauer, Daniel ENature medicine, 2019-05, Vol.25 (5), p.776-783 [Periódico revisado por pares]United States: Nature Publishing GroupTexto completo disponível |
| 3 |
Material Type: Artigo
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A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-ThalassemiaCappellini, M. Domenica ; Viprakasit, Vip ; Taher, Ali T ; Georgiev, Pencho ; Kuo, Kevin H.M ; Coates, Thomas ; Voskaridou, Ersi ; Liew, Hong-Keng ; Pazgal-Kobrowski, Idit ; Forni, G.L ; Perrotta, Silverio ; Khelif, Abderrahim ; Lal, Ashutosh ; Kattamis, Antonis ; Vlachaki, Efthymia ; Origa, Raffaella ; Aydinok, Yesim ; Bejaoui, Mohamed ; Ho, P. Joy ; Chew, Lee-Ping ; Bee, Ping-Chong ; Lim, Soo-Min ; Lu, Meng-Yao ; Tantiworawit, Adisak ; Ganeva, Penka ; Gercheva, Liana ; Shah, Farrukh ; Neufeld, Ellis J ; Thompson, Alexis ; Laadem, Abderrahmane ; Shetty, Jeevan K ; Zou, Jun ; Zhang, Jennie ; Miteva, Dimana ; Zinger, Tatiana ; Linde, Peter G ; Sherman, Matthew L ; Hermine, Olivier ; Porter, John ; Piga, AntonioThe New England journal of medicine, 2020-03, Vol.382 (13), p.1219-1231 [Periódico revisado por pares]United States: Massachusetts Medical SocietyTexto completo disponível |
| 4 |
Material Type: Artigo
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Thalassemias in South Asia: clinical lessons learnt from BangladeshHossain, Mohammad Sorowar ; Raheem, Enayetur ; Sultana, Tanvira Afroze ; Ferdous, Shameema ; Nahar, Nusrat ; Islam, Sazia ; Arifuzzaman, Mohammad ; Razzaque, Mohammad Abdur ; Alam, Rabiul ; Aziz, Sonia ; Khatun, Hazera ; Rahim, Abdur ; Morshed, ManzurOrphanet journal of rare diseases, 2017-05, Vol.12 (1), p.93-93, Article 93 [Periódico revisado por pares]England: BioMed CentralTexto completo disponível |
| 5 |
Material Type: Artigo
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Beta-thalassemiaGalanello, Renzo ; Origa, RaffaellaOrphanet journal of rare diseases, 2010-05, Vol.5 (1), p.11-11, Article 11 [Periódico revisado por pares]England: BioMed Central LtdTexto completo disponível |
| 6 |
Material Type: Artigo
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Lack of Gdf11 does not improve anemia or prevent the activity of RAP-536 in a mouse model of β-thalassemiaGuerra, Amaliris ; Oikonomidou, Paraskevi Rea ; Sinha, Sayantani ; Zhang, Jianbing ; Lo Presti, Vania ; Hamilton, Callum R. ; Breda, Laura ; Casu, Carla ; La, Ping ; Martins, Ana C. ; Sendamarai, Anoop K. ; Fleming, Mark ; Rivella, StefanoBlood, 2019-08, Vol.134 (6), p.568-572 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
| 7 |
Material Type: Artigo
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A paradigm shift on beta-thalassaemia treatment: How will we manage this old disease with new therapies?Cappellini, Maria Domenica ; Porter, John B. ; Viprakasit, Vip ; Taher, Ali T.Blood reviews, 2018-07, Vol.32 (4), p.300-311 [Periódico revisado por pares]England: Elsevier LtdTexto completo disponível |
| 8 |
Material Type: Artigo
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Clinical Classification, Screening and Diagnosis for ThalassemiaViprakasit, Vip, MD, DPhil(Oxon) ; Ekwattanakit, Supachai, MD, PhDHematology/oncology clinics of North America, 2018-04, Vol.32 (2), p.193-211 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
| 9 |
Material Type: Artigo
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α-Globin as a molecular target in the treatment of β-thalassemiaMettananda, Sachith ; Gibbons, Richard J. ; Higgs, Douglas R.Blood, 2015-06, Vol.125 (24), p.3694-3701 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
| 10 |
Material Type: Artigo
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Innovative Curative Treatment of Beta Thalassemia: Cost-Efficacy Analysis of Gene Therapy Versus Allogenic Hematopoietic Stem-Cell TransplantationCoquerelle, Séverine ; Ghardallou, Mariem ; Rais, Setti ; Taupin, Pierre ; Touzot, Fabien ; Boquet, Laure ; Blanche, Stéphane ; Benaouadi, Semir ; Brice, Thomas ; Tuchmann-Durand, Caroline ; Ribeil, Jean Antoine ; Magrin, Elisa ; Lissillour, Etienne ; Rochaix, Lise ; Cavazzana, Marina ; Durand-Zaleski, IsabelleHuman gene therapy, 2019-06, Vol.30 (6), p.753-761 [Periódico revisado por pares]United States: Mary Ann Liebert, IncTexto completo disponível |
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