标题
The gene therapy journey for hemophilia: are we there yet?
作者
关键词
-
出版物
BLOOD
Volume 120, Issue 23, Pages 4482-4487
出版商
American Society of Hematology
发表日期
2012-07-25
DOI
10.1182/blood-2012-05-423210
参考文献
相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。- Activated factor VII: my story
- (2012) U. HEDNER HAEMOPHILIA
- Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B
- (2012) Federico Mingozzi et al. MOLECULAR THERAPY
- Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide
- (2011) H. Ostergaard et al. BLOOD
- Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients
- (2011) A. D. Shapiro et al. BLOOD
- Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents
- (2011) Roberto Calcedo et al. Clinical and Vaccine Immunology
- Good Manufacturing Practice Production of Self-Complementary Serotype 8 Adeno-Associated Viral Vector for a Hemophilia B Clinical Trial
- (2011) James A. Allay et al. HUMAN GENE THERAPY
- A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8
- (2011) Virginie Monteilhet et al. MOLECULAR THERAPY
- A MicroRNA-regulated and GP64-pseudotyped Lentiviral Vector Mediates Stable Expression of FVIII in a Murine Model of Hemophilia A
- (2011) Hideto Matsui et al. MOLECULAR THERAPY
- In vivo genome editing restores haemostasis in a mouse model of haemophilia
- (2011) Hojun Li et al. NATURE
- Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges
- (2011) Federico Mingozzi et al. NATURE REVIEWS GENETICS
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Alternative Strategies for Gene Therapy of Hemophilia
- (2011) R. R. Montgomery et al. Hematology-American Society of Hematology Education Program
- Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B
- (2010) V. R. Arruda et al. BLOOD
- In vivo efficacy of platelet-delivered, high specific activity factor VIII variants
- (2010) T. K. Greene et al. BLOOD
- Assessing the potential for AAV vector genotoxicity in a murine model
- (2010) H. Li et al. BLOOD
- Codon optimization of human factor VIII cDNAs leads to high-level expression
- (2010) N. J. Ward et al. BLOOD
- Efficacy and Safety of Long-term Prophylaxis in Severe Hemophilia A Dogs Following Liver Gene Therapy Using AAV Vectors
- (2010) Denise E Sabatino et al. MOLECULAR 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
- Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses
- (2009) Roberto Calcedo et al. JOURNAL OF INFECTIOUS DISEASES
- Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial
- (2009) Albert M Maguire et al. LANCET
- Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors
- (2009) Patricia Favaro et al. MOLECULAR THERAPY
- Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
- (2009) Lajos Mátés et al. NATURE GENETICS
- X-Linked Thrombophilia with a Mutant Factor IX (Factor IX Padua)
- (2009) Paolo Simioni et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency
- (2009) Alessandro Aiuti et al. NEW ENGLAND JOURNAL OF MEDICINE
- Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX
- (2008) J. Sun et al. BLOOD
- Manufacturing and characterizing AAV-based vectors for use in clinical studies
- (2008) J F Wright GENE THERAPY
- Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
- (2008) William W. Hauswirth et al. HUMAN GENE THERAPY
- Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis
- (2008) Albert M. Maguire et al. NEW ENGLAND JOURNAL OF MEDICINE
- Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis
- (2008) James W.B. Bainbridge et al. NEW ENGLAND JOURNAL OF MEDICINE
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExploreAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now