[HTML][HTML] Functional correction of large factor VIII gene chromosomal inversions in hemophilia A patient-derived iPSCs using CRISPR-Cas9

CY Park, DH Kim, JS Son, JJ Sung, J Lee, S Bae… - Cell stem cell, 2015 - cell.com
Hemophilia A is an X-linked genetic disorder caused by mutations in the F8 gene, which
encodes the blood coagulation factor VIII. Almost half of all severe hemophilia A cases result …

Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 system

C Lyu, J Shen, R Wang, H Gu, J Zhang, F Xue… - Stem Cell Research & …, 2018 - Springer
Background Replacement therapy for hemophilia remains a lifelong treatment. Only gene
therapy can cure hemophilia at a fundamental level. The clustered regularly interspaced …

Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs

CY Park, J Kim, J Kweon, JS Son… - Proceedings of the …, 2014 - National Acad Sciences
Hemophilia A, one of the most common genetic bleeding disorders, is caused by various
mutations in the blood coagulation factor VIII (F8) gene. Among the genotypes that result in …

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

H Chen, M Shi, A Gilam, Q Zheng, Y Zhang… - Scientific reports, 2019 - nature.com
Hemophilia A is a monogenic disease with a blood clotting factor VIII (FVIII) deficiency
caused by mutation in the factor VIII (F8) gene. Current and emerging treatments such as …

CRISPR/Cas9‐mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse

Y Guan, Y Ma, Q Li, Z Sun, L Ma, L Wu… - EMBO molecular …, 2016 - embopress.org
The X‐linked genetic bleeding disorder caused by deficiency of coagulator factor IX,
hemophilia B, is a disease ideally suited for gene therapy with genome editing technology …

CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice

T Ohmori, Y Nagao, H Mizukami, A Sakata… - Scientific reports, 2017 - nature.com
Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation
factor IX gene (F9), is considered an appropriate target for genome editing technology. Here …

Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse

JP Zhang, XX Cheng, M Zhao, GH Li, J Xu, F Zhang… - Genome biology, 2019 - Springer
Background Hemophilia A, a bleeding disorder resulting from F8 mutations, can only be
cured by gene therapy. A promising strategy is CRISPR-Cas9-mediated precise insertion of …

[HTML][HTML] Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9

CJ Stephens, EJ Lauron, E Kashentseva, ZH Lu… - Journal of Controlled …, 2019 - Elsevier
Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …

CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice

C Huai, C Jia, R Sun, P Xu, T Min, Q Wang, C Zheng… - Human genetics, 2017 - Springer
Hemophilia B (HB) is an X-linked disorder caused by defects of F9 encoded coagulation
factor IX, which is an ideal model for gene therapy. Most existing HB gene therapies are …

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

L Ye, J Wang, Y Tan, AI Beyer, F Xie… - Proceedings of the …, 2016 - National Acad Sciences
Hereditary persistence of fetal hemoglobin (HPFH) is a condition in some individuals who
have a high level of fetal hemoglobin throughout life. Individuals with compound …