Publications

Functional intercellular transmission of miHTT via extracellular vesicles: an in vitro proof-of-mechanism study

Emerging Therapies for Huntington’s Disease – Focus on N‑Terminal Huntingtin and Huntingtin Exon 1

Widespread and sustained target engagement in Huntington’s disease minipigs upon intrastriatal microRNA-based gene therapy

Secreted therapeutics: monitoring durability of microRNA-based gene therapies in the central nervous system

Intrastriatal Administration of AAV5-miHTT in Non-Human Primates and Rats Is Well Tolerated and Results in miHTT Transgene Expression in Key Areas of Huntington Disease Pathology

AAV5-miHTT Gene Therapy Demonstrates Broad Distribution and Strong Human Mutant Huntingtin Lowering in a Huntington’s Disease Minipig Model

AAV5-miHTT Gene Therapy Demonstrates Sustained Huntingtin Lowering and Functional Improvement in Huntington Disease Mouse Models

Translation of MicroRNA-Based Huntingtin-Lowering Therapies from Preclinical Studies to the Clinic

Potent and Sustained Huntingtin Lowering via AAV5 Encoding miRNA Preserves Striatal Volume & Cognitive Function in a Humanized Mouse Model of HD

AAV5-miHTT Lowers Huntingtin mRNA and Protein without Off-Target Effects in Patient-Derived Neuronal Cultures and Astrocytes

Design, Characterization, and Lead Selection of Therapeutic miRNAs Targeting Huntington for Development of Gene Therapy for Huntington’s Disease

AAV5-miHTT gene therapy demonstrates suppression of mutant huntingtin aggregation and neuronal dysfunction in a rat model of Huntington’s disease

GluK2 Is a Target for Gene Therapy in Drug-Resistant Temporal Lobe Epilepsy

Hemophilia Gene Therapy Value Assessment: Methodological Challenges and Recommendations

Evidence of a disability paradox in patient-reported outcomes in haemophilia

Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

Adult lifetime cost of hemophilia B management in the U.S.: Payer and societal perspectives from a decision analytic model

Health care resource utilization and cost burden of hemophilia B in the United States

Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B

Gene Therapy with Adeno-Associated Virus Vector 5‑Human Factor IX in Adults with Hemophilia B

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs

Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates

Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5ch and AAV1

PhP.B Enhanced Adeno-Associated Virus Mediated-Expression Following Systemic Delivery or Direct Brain Administration

Transduction patterns in the CNS following various routes of AAV-5-mediated gene delivery

Optimization of viral protein ratios for production of rAAV serotype 5 in the baculovirus system

MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain

Insect Cell-Based Recombinant Adeno-Associated Virus Production: Molecular Process Optimization

Phase I Open Label Liver-Directed Gene Therapy Clinical Trial for Acute Intermittent Porphyria

From mutation identification to therapy: Discovery and origins of Glybera, the first approved gene therapy in the Western World