In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy

Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver

(2021) M. Kim et al.   Science Advances

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2021) Jeong Pil Han et al.   Biology-Basel

Hemophilia therapy: the future has begun

(2020) Pier Mannuccio Mannucci   HAEMATOLOGICA

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing

(2020) Qiang Cheng et al.   Nature Nanotechnology

Synergistic lipid compositions for albumin receptor mediated delivery of mRNA to the liver

(2020) Lei Miao et al.   Nature Communications

Lipid nanoparticle technology for therapeutic gene regulation in the liver

(2020) Dominik Witzigmann et al.   ADVANCED DRUG DELIVERY REVIEWS

Spectroscopic Evaluation of Red Blood Cells of Thalassemia Patients with Confocal Microscopy: A Pilot Study

(2020) Laura Rey-Barroso et al.   SENSORS

Latest Developed Strategies to Minimize the Off-Target Effects in CRISPR-Cas-Mediated Genome Editing

(2020) Muhammad Naeem et al.   Cells

CRISPR Gene Therapy: Applications, Limitations, and Implications for the Future

(2020) Fathema Uddin et al.   Frontiers in Oncology

Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

(2019) Morgan L. Maeder et al.   NATURE MEDICINE

Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing

(2019) Danny Wilbie et al.   ACCOUNTS OF CHEMICAL RESEARCH

Timing of inhibitor development in more than 1000 previously untreated patients with severe hemophilia A

(2019) H. Marijke van den Berg et al.   BLOOD

Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: Phase 2 trial results

(2019) Amy D. Shapiro et al.   BLOOD

Hemophilia A and B: molecular and clinical similarities and differences

(2019) Giancarlo Castaman et al.   HAEMATOLOGICA

Development and Clinical Translation of Approved Gene Therapy Products for Genetic Disorders

(2019) Alireza Shahryari et al.   Frontiers in Genetics

The Development of Functional Non-Viral Vectors for Gene Delivery

(2019) Patil et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Immunogenicity of Cas9 Protein

(2019) Aditi Mehta et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Advances and innovations in haemophilia treatment

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

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

(2018) Jonathan D. Finn et al.   Cell Reports

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

Directed evolution of CRISPR-Cas9 to increase its specificity

(2018) Jungjoon K. Lee et al.   Nature Communications

Cas9 immunity creates challenges for CRISPR gene editing therapies

(2018) Julie M. Crudele et al.   Nature Communications

Prevalence of and risk factors for cerebral microbleeds among adult patients with haemophilia A or B

(2017) H. Husseinzadeh et al.   HAEMOPHILIA

Enhanced proofreading governs CRISPR–Cas9 targeting accuracy

(2017) Janice S. Chen et al.   NATURE

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

(2017) Hao Yin et al.   NATURE BIOTECHNOLOGY

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

(2017) Johannes Oldenburg et al.   NEW ENGLAND JOURNAL OF MEDICINE

Adeno-Associated Virus Gene Therapy for Liver Disease

(2016) Lisa M. Kattenhorn et al.   HUMAN GENE THERAPY

The past and future of haemophilia: diagnosis, treatments, and its complications

(2016) Flora Peyvandi et al.   LANCET

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

(2016) Yong Wu et al.   Scientific Reports

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

(2015) R. Sharma et al.   BLOOD

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Digenome-seq: genome-wide profiling of CRISPR-Cas9 off-target effects in human cells

(2015) Daesik Kim et al.   NATURE METHODS

Targeting Antithrombin to Treat Hemophilia

(2015) Margaret V. Ragni   NEW ENGLAND JOURNAL OF MEDICINE

Incidence, mortality rates and causes of deaths in haemophilia patients in Sweden

(2013) S. Lövdahl et al.   HAEMOPHILIA

Endogenous factor VIII synthesis from the intron 22–inverted F8 locus may modulate the immunogenicity of replacement therapy for hemophilia A

(2013) Gouri Shankar Pandey et al.   NATURE MEDICINE

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer

(2012) G. Buchlis et al.   BLOOD

Surgical Experience with rFVIIa (NovoSeven) in congenital haemophilia A and B patients with inhibitors to factors VIII or IX

(2011) L. A. VALENTINO et al.   HAEMOPHILIA

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

(2010) Sylvie Boutin et al.   HUMAN GENE THERAPY

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

The Haemostatic Role of Tissue Factor Pathway Inhibitor

(2007) James T.B. Crawley et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY