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| 1 | CRISPR-Cas9基因编辑用于镰状细胞病和β地中海贫血的治疗显示文摘输血依赖性β地中海贫血(transfusion-dependent β-thalassemia,TDT)和镰状细胞病(sickle cell disease,SCD)是全球最常见的单基因疾病。SCD是血红蛋白的遗传缺陷,会导致红细胞变成月牙形。这些细胞可以溶解并阻塞小血管,从而剥夺人体组织中的氧气。该疾病可引起极度疼痛并损害肺、心脏、肾脏和肝脏。β地中海贫血是一种血液疾病,可减少血红蛋白的产生。 | 张四维(编译) Frangoul H Altshuler D Cappellini MD | 2021 | 国际老年医学杂志2021，42，5： | 3 |
| 2 | Seven novel point mulations in the uroporphyrinogen decarboxylase (UROD) gene in patients with familial porphyria cutanea larda (f-PCT) 显示文摘 | Cappellini MD Martinez di Montemuros F Tavazzi D | 2001 | Hum Murat2001，17，4： | 1 |
| 3 | A phase 3 study of deferasirox(ICL670) ,a once - daily oral iron chelator, in patients with β- thalassemia显示文摘 | Cappellini MD Cohen A Piga A | 2006 | Blood2006，107，9： | 1 |
| 4 | Thalassemia intermedia:revisited显示文摘 | Taher A Isma'eel H Cappellini MD | 2006 | Blood Cells Mol Dis2006，37，： | 1 |
| 5 | Serum ferritin level and morbidity risk in transfusion-independent patients with beta-thalassemia intermedia:the ORIENT study显示文摘 | Musallam KM Cappellini MD Daar S | 2014 | Haematologica2014，99，： | 1 |
| 6 | Recent advances and treatment challenges in patients with non-transfusion-dependent thalassemia显示文摘 | Taher AT Cappellini MD Musallam KM | 2012 | Blood Rev2012，26，1： | 1 |
| 7 | Elevated liver iron concentration is a marker of increased morbidity in patients with beta thalassemia intermedia显示文摘 | Musallam KM Cappellini MD Wood JC | 2011 | Haematologica2011，96，： | 1 |
| 8 | Overcoming the challenge of patient compliance with iron emin显示文摘 | Cappellini MD | 2005 | Hematol2005，42，： | 1 |
| 9 | A phase 3 study of defera?sirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia显示文摘 | Cappellini MD Cohen A Piga A | 2006 | Blod2006，107，9： | 1 |
| 10 | Neridronate improves bone mineral density and reduces back pain in β-thalassaemia patients with osteoporosis:results from a phase 2,randomized,parallel-arm,open-label study显示文摘 | Forni GL Perrotta S Giusti A Quarta G Pitrolo L Cappellini MD D'Ascola DG Borgna Pignatti C Rigano P Filosa A Iolascon G Nobili B Baldini M Rosa A Pinto V Palummeri E | | 0，，： | 1 |
| 11 | Glucose -6 -phosphate dehydrogen- ase deficiency 显示文摘 | Cappellini MD Fiorelli G | 2008 | Lancet2008，371，9606： | 1 |
| 12 | An update on thalassemia intermedia显示文摘 | Maakaron JE Cappellini MD Taher AT | 2013 | J Med Liban2013，61，3： | 1 |
| 13 | Long-term experience with deferasirox (ICL670), a once-daily oral iron chelator, in the treatment of transfusional iron overload 显示文摘 | Cappellini MD Taher A | 2008 | Expert Opin Pharmacother2008，9，： | 1 |
| 14 | A reappraisal of Gaucher dis- ease-diagnosis and disease management algorithms 显示文摘 | Mistry PK Cappellini MD Lukina E | 2011 | Am J Hema- tol2011，86，1： | 1 |
| 15 | glucose-6-phosphate dehydro- genase deficiency 显示文摘 | Cappellini MD Fiorelli G | 2008 | Lancet2008，，371： | 1 |
| 16 | Redefining thalassemia as a hypercoagulable state显示文摘 | Cappellini MD Motta I Musallam KM | 2010 | Ann N Y Acad Sci2010，1202，： | 1 |
| 17 | Phase Ⅱ study of oral chelator ICL670 in the thalassaemia patients with transfusional iron overload: efficacy, safety, pharmacokinetics (PK) and pharmacodynamics(PD) after 6months of therapy显示文摘 | PIGA A GALANELLO R CAPPELLINI MD | 2002 | Blood2002，100，11： | 1 |
| 18 | Iron-chelating therapy with the new oral agent ICL670 (Exjade) 显示文摘 | CAPPELLINI MD | 2005 | Best Pract Res Clin Haematol2005，18，2： | 1 |
| 19 | A phase 3 study of defer- asirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia显示文摘 | Cappellini MD Cohen A Piga A | 2006 | Blood2006，107，9： | 1 |
| 20 | A reappraisal of Gaucher disease diagnosis and disease management algorithms 显示文摘 | Mistry PK Cappellini MD Lukina E | 2011 | Am J Hc- matol2011，86，1： | 1 |
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