Universal Approach to Correct Various HBB Gene Mutations in …
2017年11月21日 · Using human induced pluripotent stem cells (iPSCs) from two β-thal patients with different HBB gene mutations, we devised and tested a universal strategy to achieve …
Development of β-globin gene correction in human ... - Science
SCD is an autosomal recessive disease resulting from a single point mutation in codon six of the β-globin gene (HBB). Ex vivo β-globin gene correction in autologous patient-derived …
A Universal Approach to Correct Various HBB Gene
2017年12月8日 · Using human induced pluripotent stem cells (iPSCs) from two β-thalassemia patients with different HBB gene mutations, we devised and tested a universal strategy to …
Gene replacement of α-globin with β-globin restores ... - Nature
2021年3月18日 · Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1 gene with a full-length HBB transgene in β-thalassemia-derived …
A Universal Approach to Correct Various HBB Gene Mutations in …
Using human induced pluripotent stem cells (iPSCs) from two β-thal patients with different HBB gene mutations, we devised and tested a universal strategy to achieve targeted insertion of …
Development of gene editing strategies for human β-globin (HBB…
2020年4月15日 · SCD is caused by a point mutation in human β-globin gene (HBB). Clinical strategies have demonstrated substantial success, however there is not any permanent cure …
Preclinical Development of HBB Gene Correction in Autologous ...
2017年12月7日 · Here we present our IND-enabling preclinical development for CRISPR-Cas9 and rAAV6-mediated HBB genome editing of the E6V mutation in SCD-derived HSPCs as well …
A Previously Undescribed β+ Thalassemia Mutation (HBB:c.
2020年7月18日 · Hemoglobin profile was defined by IEF and CE-HPLC procedures. Identification of the β-globin genotype was carried out by PCR and nucleotide sequencing of the β-globin …
Seamless correction of the sickle cell disease mutation of the HBB …
Sickle cell disease (SCD) is the most common human genetic disease which is caused by a single mutation of human β-globin (HBB) gene. The lack of long-term treatment makes the …
β-血红蛋白病的基因治疗临床试验进展_南方+_南方plus
2023年11月23日 · β-血红蛋白病是一类由于编码β珠蛋白的β-珠蛋白基因（HBB）基因致病变异引起的疾病，包括β-地中海贫血症及镰状细胞病（sickle cell disease，SCD）等，是目前世界上 …