Novel Severe Hemophilia A Mouse Model with Factor VIII Intron 22 Inversion

The Immune Response to the fVIII Gene Therapy in Preclinical Models

(2020) Seema R. Patel et al.   Frontiers in Immunology

F8 Inversions at Xq28 Causing Hemophilia A Are Associated With Specific Methylation Changes: Implication for Molecular Epigenetic Diagnosis

(2019) Muhammad Ahmer Jamil et al.   Frontiers in Genetics

Advances and innovations in haemophilia treatment

(2018) Rob Peters et al.   NATURE REVIEWS DRUG DISCOVERY

AAV: An Overview of Unanswered Questions

(2017) Kenneth I. Berns et al.   HUMAN GENE THERAPY

Cas-analyzer: an online tool for assessing genome editing results using NGS data

(2016) Jeongbin Park et al.   BIOINFORMATICS

In situ genetic correction of F8 intron 22 inversion in hemophilia A patient-specific iPSCs

(2016) Yong Wu et al.   Scientific Reports

Clinical characterization of int22h1/int22h2-mediated Xq28 duplication/deletion: new cases and literature review

(2015) Ayman W El-Hattab et al.   BMC Medical Genetics

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9

(2015) Chul-Yong Park et al.   Cell Stem Cell

Efficient inversions and duplications of mammalian regulatory DNA elements and gene clusters by CRISPR/Cas9

(2015) Jinhuan Li et al.   Journal of Molecular Cell Biology

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

(2014) C.-Y. Park et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Renal diseases in haemophilic patients: pathogenesis and clinical management

(2013) Pasquale Esposito et al.   EUROPEAN JOURNAL OF HAEMATOLOGY

F8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysis

(2012) S. C. Gouw et al.   BLOOD

Guidelines for the management of hemophilia

(2012) A. Srivastava et al.   HAEMOPHILIA

Targeted chromosomal duplications and inversions in the human genome using zinc finger nucleases

(2011) H. J. Lee et al.   GENOME RESEARCH

Influence of the type of F8 gene mutation on inhibitor development in a single centre cohort of severe haemophilia A patients

(2010) S. C. GOUW et al.   HAEMOPHILIA