AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

Hemophilia Gene Therapy: A Holy Grail Found

(2011) Katherine P Ponder   MOLECULAR THERAPY

A Cell-penetrating Peptide Suppresses Inflammation by Inhibiting NF-κB Signaling

(2011) Yu Fu Wang et al.   MOLECULAR THERAPY

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII

(2011) Chuhong Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

(2010) J. D. Finn et al.   BLOOD

Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction

(2010) Nuria Carrillo-Carrasco et al.   HUMAN GENE THERAPY

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

RH10 provides superior transgene expression in mice when compared with natural AAV serotypes for neonatal gene therapy

(2010) Chuhong Hu et al.   JOURNAL OF GENE MEDICINE

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

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

(2010) Kevin D Foust et al.   NATURE BIOTECHNOLOGY

Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model

(2009) D. E. Sabatino et al.   BLOOD

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

(2009) Randy J Chandler et al.   MOLECULAR THERAPY

Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models

(2009) Lili Wang et al.   MOLECULAR THERAPY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

Evaluation of Gene Promoters for Liver Expression by Hydrodynamic Gene Transfer

(2008) Andrew T. Nguyen et al.   JOURNAL OF SURGICAL RESEARCH

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY