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Journal Articles

No CpGs for AAVs?

Lindsey A. George

Blood (2021) 137 (6): 721–723.

https://doi.org/10.1182/blood.2020009285

Published: 2021

Journal Articles

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Steven Pipe, MD, Michael Becka, Elke Detering, Konstantina Vanevski, Toshko Lissitchkov

Blood (2019) 134 (Supplement_1): 4630.

https://doi.org/10.1182/blood-2019-125764

Published: 2019

Journal Articles

Proteasome Inhibitors Impair the Innate Antiviral Immune Response and Potentiate Pelareorep-Based Viral Therapy in Multiple Myeloma

Ada Dona', PhD, Domenico Viola, Enrico Caserta, PhD, Francesca Besi, PhD, James F Sanchez, PhD, Guido Marcucci, MD, Jonathan J Keats, PhD, Amrita Y. Krishnan, MD, Matt Coffey, PhD, Douglas W. Sborov, MDMSc, Gerard Nuovo, MD, Craig C Hofmeister, MDMPH, Flavia Pichiorri, PhD

Blood (2019) 134 (Supplement_1): 1816.

https://doi.org/10.1182/blood-2019-128740

Published: 2019

Journal Articles

Correcting Rare Blood Disorders Using Coagulation Factors Produced In Vivo By Shielded Living Therapeutics TM Products

Guillaume Carmona, PhD, Lauren Barney, PhD, Jared Sewell, PhD, Ryan Newman, Christine Carroll, Michael Beauregard, Janet Huang, MSc, Richard W Heidebrecht, Jr., PhD, Deya Corzo, MD, David Moller, MD, Devyn Smith, PhD, David Peritt, PhD, Rogerio Vivaldi, MD MBA

Blood (2019) 134 (Supplement_1): 2065.

https://doi.org/10.1182/blood-2019-127050

Published: 2019

Journal Articles

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Wolfgang Miesbach, MD PhD, Karina Meijer, Michiel Coppens, MD, Peter Kampmann, MD, Dr. Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, MD, Erhard Seifried, MD PhD, Joachim Schwaeble, Halvard Bönig, Eileen K Sawyer, PhD, Frank W.G. Leebeek, MD PhD

Blood (2019) 134 (Supplement_1): 2059.

https://doi.org/10.1182/blood-2019-122535

Published: 2019

Journal Articles

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

Blood (2019) 134 (Supplement_1): 4638.

https://doi.org/10.1182/blood-2019-128490

Published: 2019

Journal Articles

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Anita Kruzik, PhD, Roland Raim, PhD, Dirk Voelkel, PhD, Markus Weiller, PhD, Werner Hoellriegl, PhD, Friedrich Scheiflinger, Hanspeter Rottensteiner, Birgit M Reipert, PhD, Maurus de la Rosa

Blood (2019) 134 (Supplement_1): 5922.

https://doi.org/10.1182/blood-2019-126730

Published: 2019

Journal Articles

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Nusrat Khan, MSc, Sridhar Bammidi, Jayandharan Giridhara Rao, PhD

Blood (2019) 134 (Supplement_1): 3351.

https://doi.org/10.1182/blood-2019-125222

Published: 2019

Journal Articles

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Lindsey A. George, MD, Spencer K. Sullivan, MD, John E.J. Rasko, MBBS, PhD, Adam Giermasz, MD PhD, Benjamin J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD MPH, Jerome M. Teitel, MD FRCPC, Catherine E. McGuinn, MD, Alexa R. Runowski, MS, Fraser Wright, PhD, Xavier M. Anguela, PhD, Katherine A. High, MD, Denis Rybin, PhD, John E. Murphy, PhD, Jeremy Rupon, MD

Blood (2019) 134 (Supplement_1): 3347.

https://doi.org/10.1182/blood-2019-124091

Published: 2019

Journal Articles

Liver-Directed AAV Gene Therapy for Gaucher Disease

Carlos J Miranda, PhD, Miriam Canavese, PhD, Elisa Chisari, MS, Jalpa Pandya, MPhil, Clement Cocita, PhD, Maria Portillo, MRes, Jenny McIntosh, PhD, Azadeh Kia, PhD, Jonathan H. Foley, PhD, Allison Dane, PhD, Jey M Jeyakumar, PhD, Rose Sheridan, PhD, Romuald Corbau, Amit C. Nathwani, MD PhD

Blood (2019) 134 (Supplement_1): 3354.

https://doi.org/10.1182/blood-2019-124280

Published: 2019

Journal Articles

Update on clinical gene therapy for hemophilia

George Q. Perrin, Roland W. Herzog, David M. Markusic

Blood (2019) 133 (5): 407–414.

https://doi.org/10.1182/blood-2018-07-820720

Published: 2019

Journal Articles

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Frank Leebeek, K Meijer, MD, Michiel Coppens, MD, Peter Kampmann, MD, Robert Klamroth, Roger Schutgens, MD PhD MSc, Giancarlo Castaman, Erhard Seifried, MD, J Schwaeble, MD, Halvard Bonig, MD, Eileen K Sawyer, PhD, W Miesbach

Blood (2018) 132 (Supplement 1): 3476.

https://doi.org/10.1182/blood-2018-99-109995

Published: 2018

Journal Articles

Evidence for Epitope-Specific T Cell Responses in HIV-Associated Non Neoplastic Lymphadenopathy: High-Throughput Immunogenetic Evidence

Katerina Gemenetzi, Evangelia Stalika, Andreas Agathangelidis, PhD, Fotis Psomopoulos, Elisavet Vlachonikola, Chrysi Galigalidou, Symeon Metallidis, Triantafylia Koletsa, Kostas Stamatopoulos, MD PhD, Maria Papaioannou, Anastasia Hadzidimitriou, PhD

Blood (2018) 132 (Supplement 1): 1117.

https://doi.org/10.1182/blood-2018-99-118975

Published: 2018

Journal Articles

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

Amit C Nathwani, MD PhD, Ulrike Reiss, MD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Anne Riddell, Jun Pie, Johnny N Mahlangu, MD, Michael Recht, MD PhD, Yu-Min Shen, MD, Kathleen G. Halka, MD, Michael M Meagher, PhD, Arthur W. Nienhuis, MD, Andrew M. Davidoff, MD, Sarah Mangles, Chris L Morton, Zhou Junfang, Vlad Calin Radulescu, MD

Blood (2018) 132 (Supplement 1): 491.

https://doi.org/10.1182/blood-2018-99-118334

Published: 2018

Journal Articles

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Blood (2018) 132 (Supplement 1): 487.

https://doi.org/10.1182/blood-2018-99-115495

Published: 2018

Journal Articles

An International Epidemiology Study to Determine the Prevalence of Preexisting Cell-Mediated Immunity to Adeno-Associated Virus (AAV) in Adults with Severe Hemophilia

Kavitha S. Rajavel, PhD, John C. Chapin, MD, Ying Tang

Blood (2018) 132 (Supplement 1): 5794.

https://doi.org/10.1182/blood-2018-99-113308

Published: 2018

Journal Articles

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Amit C Nathwani, MD PhD, Edward Tuddenham, MD, Pratima Chowdary, MD, Jenny McIntosh, PhD, Doyoung Lee, PhD, Cecilia Rosales, PhD, Mark Phillips, Jun Pie, Zhou Junfang, Michael M Meagher, PhD, Ulrike Reiss, MD, Andrew M. Davidoff, MD, Chris L Morton, Anne Riddell

Blood (2018) 132 (Supplement 1): 489.

https://doi.org/10.1182/blood-2018-99-118256

Published: 2018

Journal Articles

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Azadeh Kia, PhD, Jenny McIntosh, PhD, Cecilia Rosales, PhD, Paniz Hosseini, Rose Sheridan, Justyna Spiewak, Kevin Mills, Romuald Corbau, PhD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2209.

https://doi.org/10.1182/blood-2018-99-116632

Published: 2018

Journal Articles

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

Allison Dane, PhD, Jenny McIntosh, Doyoung Lee, PhD, Rose Sheridan, Mark Maginn, Romuald Corbau, PhD, Andrew M. Davidoff, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 2197.

https://doi.org/10.1182/blood-2018-99-117969

Published: 2018

Journal Articles

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

Pratima Chowdary, MD, Susie Shapiro, MD, Andrew M. Davidoff, MD, Ulrike Reiss, MD, Rebecca Alade, PhD, Geoff Brooks, BSc, Allison Dane, PhD, Jenny McIntosh, PhD, Gerard Short, MBBS, Edward Tuddenham, MD, Amit C Nathwani, MD PhD

Blood (2018) 132 (Supplement 1): 631.

https://doi.org/10.1182/blood-2018-99-118050

Published: 2018

NARROW

No CpGs for AAVs?

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A

Proteasome Inhibitors Impair the Innate Antiviral Immune Response and Potentiate Pelareorep-Based Viral Therapy in Multiple Myeloma

Correcting Rare Blood Disorders Using Coagulation Factors Produced In Vivo By Shielded Living Therapeutics TM Products

Stable FIX Expression and Durable Reductions in Bleeding and Factor IX Consumption for up to 4 Years Following AMT-060 Gene Therapy in Adults with Severe or Moderate-Severe Hemophilia B

Design and Characterization of FLT210, a Potent Next Generation AAV-hFVIII Vector Candidate

AAV8-Specific Immune Adsorption Column: A Treatment Option for Patients with Pre-Existing Anti-AAV8 Neutralizing Antibodies

Suicide Gene Therapy with a CD33 Targeted AAV6 Vector Expressing an Inducible Caspase-9 Suicide Gene Is Therapeutic in a Xenotransplantation Model of Acute Myeloid Leukemia

Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year

Liver-Directed AAV Gene Therapy for Gaucher Disease

Update on clinical gene therapy for hemophilia

Reduction in Annualized Bleeding and Factor IX Consumption up to 2.5 Years in Adults with Severe or Moderate-Severe Hemophilia B Treated with AMT-060 (AAV5-hFIX) Gene Therapy

Evidence for Epitope-Specific T Cell Responses in HIV-Associated Non Neoplastic Lymphadenopathy: High-Throughput Immunogenetic Evidence

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

An International Epidemiology Study to Determine the Prevalence of Preexisting Cell-Mediated Immunity to Adeno-Associated Virus (AAV) in Adults with Severe Hemophilia

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Preclinical Evaluation of an Engineered AAV Capsid in Non-Human Primates for the Treatment of Haemophilia B

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

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ASH Publications

American Society of Hematology

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ASH Publications

American Society of Hematology

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