Blood. 2016;128:121.Swift R, Abdulmalik O, Chen Q, et al. SCD-101: A new anti-sickling drug reduces pain and fatigue and improves red blood cell shape in peripheral blood of patients with sickle cell disease. Blood;128(22):121....
镰刀型细胞贫血病(Sickle Cell Disease,SCD)又称为镰状细胞病、镰状细胞贫血(SCA),是 […] 镰刀型细胞贫血病(Sickle Cell Disease,SCD)又称为镰状细胞病、镰状细胞贫血(SCA),是一种常染色体显性遗传血红蛋白病,因β-肽链第6位氨基酸谷氨酸被缬氨酸所代替,构成镰状血红蛋白,取代了正常血红蛋白。临床表现为慢性溶血...
请注意这一点,因为这给后来的治疗方案之一买下了伏笔。SCD患者可能会出现多项健康问题,例如突发的疼痛(镰刀型贫血危机,sickle-cell crisis),贫血,细菌感染和中风。SCD患者出现的疼痛非同寻常,有些人形容为好比不停的被刀捅,还有些人说就好像全身发生了好几处骨折。当患者年纪稍长之后可能会出现慢性疼痛。发达国家...
Sickle-cell anemia / Sickle cell anemia / Sickle cell-hemoglobin SS disease (disorder) / Anemia sickle cell / Anemia, Sickle Cell / Sickle-cell/HB-C disease / Sickle-cell/Hb-C disease without crisis / Hemoglobin SC Disease / Sickle cell-hemoglobin C disease (disorder) / Sickle cell Hb-C...
BEAM-101通过在体外对患者的造血干细胞进行基因组改造,使其模仿遗传性持续性胎儿血红蛋白症个体中的单核苷酸多态性,来缓解导致SCD的遗传突变的不良影响。BEAM-101于2021年11月获得了FDA的IND批准。和其他体外基因编辑疗法一样,BEAM-101也面临着与化疗清髓相关的毒性,为此,Beam公司的下一波SCD体外项目将对细胞的编辑方...
Taylor VI, MD (Howard University School of Medicine); Marsha Treadwell, PhD (UCSF Benioff Children’s Hospital); Cassandra Trimnell (Sickle Cell 101); Derek van Amerongen, MD, MS (Humana of Ohio); Heidi Wagner, JD (Global Blood Therapeutics); Shamonica Wiggins....
About Sickle Cell Disease Sickle cell disease (SCD) is a genetic disorder of the red blood cells caused by a mutation in the HBB gene. This gene encodes a protein that is a key component of hemoglobin, a protein complex whose function is to transport oxygen in the body. The res...
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease Blood, 101 (6) (2003), pp. 2137-2143 View PDFView articleView in ScopusGoogle Scholar [31] F. Bernaudin, G. Socie, M. Kuentz, et al. Long-term results of related myeloablative stem cell trans...
Hematol. 2002, 9, 101–106. [CrossRef] [PubMed] 3. Gill, F.M.; Sleeper, L.A.; Weiner, S.J.; Brown, A.K.; Bellevue, R.; Grover, R.; Pegelow, C.H.; Vichinsky, E. Clinical events in the first decade in a cohort of infants with sickle cell disease. Cooperative Study of...
The investment firm William Blair see some early signs of differentiation for BEAM-101. In a note sent to investors, analyst Sami Corwin said the Beam therapy’s ability to elicit higher fetal hemoglobin levels, resulting in lower sickle cell hemoglobin, could translate into extended VOC-free in...