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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 | Safety of high dose trivalent inactivated influenza vaccine in pediatric patients with acute lymphoblastic leukemia显示文摘 | McManus Mv Frangoul H McCullers JA et a1 | 2014 | Pediatr Blood Cancer2014，61，5： | 1 |
| 3 | Comparison of phannacokinetics and safety of voriconazole intravenous-to-oral switch in immunocompromised children and healthy adults显示文摘 | DRISCOLL TA YU L C FRANGOUL H | 2011 | Antimicrob Agents Chemother2011，55，12： | 1 |
| 4 | Acyclovir - resistant herpes simplex virus pneumonia post - unrelated stem cell transplantation : a word of caution显示文摘 | Frangoul H Wills M Crossno C | 2007 | Pediatr Transplant2007，11，： | 1 |
| 5 | Two novel CHS1 (LYST) mutations : clinical correlations in an infant with Chediak-Higashi syndrome 显示文摘 | Zarzour W Kleta R Frangoul H | 2005 | Mol Genet Metab2005，85，2： | 1 |
| 6 | A cord blood transplant recipient with Mycobacterium mueogenieurn central venous catheter infection after infusion of tap water显示文摘 | Fleming GA Frangoul H Dermody TS | 2006 | Pediatr Infect Dis2006，25，6： | 1 |
| 7 | Preengraflment syndrome after unre- lated cord blood transplant显示文摘 | Frangoul H Wang L Ho R | 2009 | Biol Blood Marrow Transplant2009，15，11： | 1 |
| 8 | Castleman disease in a pediatric liver transplant recipient: A case report and literature review显示文摘 | Hugo J. R. Bonatti Jason Axt Ellen Bailey Hunter Sarah Louise Lott Haydar Frangoul Lynette Gillis Hernan Correa Beau Kelly | 2011 | Pediatric Transplantation2011，，6： | 1 |
| 9 | Etanercept for treatment of idiopathic pneumonia syndrome after allogeneic hematopoietic stem cell transplantation 显示文摘 | Frangoul H Koyama T Domm J | 2009 | Blood2009，113，12： | 1 |
| 10 | Two novel CHS1 (LYST) mutations:clinical correlations in an infant with Chediak-Higashi syndrome显示文摘 | Zarzour W Kleta R Frangoul H | 2005 | Mol Genet Metab2005，85，： | 1 |
| 11 | Two novel CHS(LYST) mutations:clinical correlations in an infant with Chediak-Higashi syndrome显示文摘 | Zarzour W Kleta R Frangoul H | 2005 | Mol Fenet Metab2005，85，2： | 1 |
| 12 | Allogeneic bone marrow transplantation in children with myelodysplastic syndrome or juvenile myelomonocytic leukemia: the Seattle experience显示文摘 | Yusuf U Frangoul HA Gooley TA | 2004 | Bone Marrow Transplant2004，33，8： | 1 |
| 13 | Allergenic bone mar- row transplantation in children with myelodysplastie syndrome or juvenile myelomonocytic leukemia: the Seattle experience 显示文摘 | Yusuf U Frangoul HA Gooley TA | 2004 | Bone Marrow Transpl2004，33，： | 1 |
| 14 | Phase I Study ofTopotecan Administered as a 21-Day Continuous Infusion inChildren with Recurrent Solid Tumors; A Report from theChildren's Cancer Group 显示文摘 | Frangoul H Ames MM Mosher RB | 1999 | Clin Cancer Res1999，5，12： | 1 |
| 15 | Comparison of pharmacokinetics and safety of voriconazole intravenous-to-oral switch in immunocompromised adolescents and healthy adults显示文摘 | Driscoll TA Frangoul H Nemecek ER | 2011 | Antimicrob Agents Chemother2011，55，12： | 1 |
| 16 | Compari- son of pharmacokinetics and safety of voriconazole intrave-nous-to-oral switch in immunocompromised children and healthy adults显示文摘 | DRISCOLL T A YU L C FRANGOUL H | 2011 | Antimicrob Agents Chemother2011，55，12： | 1 |
| 17 | Comparison of pharmacokinetics and safety of voriconazole intravenous-to-oral switch in immunocompromised adolescents and healthy adults 显示文摘 | Driscoll TA Frangoul H Nemecek ER | 2011 | Antimicrob Agents Chemother2011，55，12： | 1 |
| 18 | Effects of total body irradiation (TBI) dosing on pulmonary toxicity and treatment related mortality in pediatric stem cell rransplant (SCT) recipients 显示文摘 | Tenenholz T Guttman S Frangoul H | 2007 | Int J Radiat Oncol Biol Phys2007，69，3： | 1 |
| 19 | A prospective study of G-CSF primed bone marrow as a stem cell course for allogeneic bone marrow transplant in pediatrics : a Pediatric Blood and Marrow Transplant Consortium ( PBMTC ) study 显示文摘 | Frangoul H Nemecek ER Billheimer D | 2007 | Blood2007，110，： | 1 |
| 20 | CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia显示文摘Transfusion-dependent β-thalassemia(TDT)and sickle cell disease(SCD)are severe monogenic diseases with severe and potentially life-threatening manifestations.BCL11A is a transcription factor that represses γ-globin expression and fetal hemoglobin in erythroid cells.We performed electroporation of CD34+hematopoietic stem and progenitor cells obtained from healthy donors,with CRISPR-Cas9 targeting the BCL11A erythroid-specific enhancer.Approximately 80% of the alleles at this locus were modified,with no evidence of off-target editing.After undergoing myeloablation,two patients-one with TDT and the other with SCD-received autologous CD34+cells edited with CRISPR-Cas9 targeting the same BCL11A enhancer.More than a year later,both patients had high levels of allelic editing in bone marrow and blood,increases in fetal hemoglobin that were distributed pancellularly,transfusion independence,and(in the patient with SCD)elimination of vaso-occlusive episodes. | Haydar Frangoul | 2020 | 四川生理科学杂志2020，42，4： | 0 |
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