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Journal Articles

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Sylvia von Mackensen, Jonathan M. Ducore, Lindsey A. George, Adam Giermasz, Catherine McGuinn, John E. J. Rasko, Ben J Samelson-Jones, Spencer K. Sullivan, Jerome M. Teitel, Amit Chhabra, Annie F. Fang, Amanda O'Brien, Frank Plonski, Jeremy Rupon, Lynne Smith, Ian Winburn

Blood (2023) 142 (Supplement 1): 3628.

https://doi.org/10.1182/blood-2023-179431

Published: 2023

Journal Articles

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Ben J. Samelson-Jones, Spencer K. Sullivan, John E.J. Rasko, Adam Giermasz, Lindsey A. George, Jonathan M. Ducore, Jerome M. Teitel, Catherine E. McGuinn, Amanda O'Brien, Ian Winburn, Lynne M Smith, Amit Chhabra, Jeremy Rupon

Blood (2021) 138 (Supplement 1): 3975.

https://doi.org/10.1182/blood-2021-150541

Published: 2021

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

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

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Blood (2017) 130 (Supplement 1): 601.

https://doi.org/10.1182/blood.V130.Suppl_1.601.601

Published: 2017

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Daniel J Hui, PhD BS, Yun Liu, PhD, Ruchi Patel, Yifeng Chen, MD, Lindsey A. George, MD, Spencer K. Sullivan, MD, Adam Giermasz, MD, John Rasko, MBBS, PhD, Ben J. Samelson-Jones, MD PhD, Jonathan M. Ducore, MD, Sue Dasen, MS, Marcus E. Carr, MD PhD, Xavier M. Anguela, PhD, Katherine A. High, MD

Blood (2017) 130 (Supplement 1): 2056.

https://doi.org/10.1182/blood.V130.Suppl_1.2056.2056

Published: 2017

Journal Articles

FLI1 level during megakaryopoiesis affects thrombopoiesis and platelet biology

Karen K. Vo, Danuta J. Jarocha, Randolph B. Lyde, Vincent Hayes, Christopher S. Thom, Spencer K. Sullivan, Deborah L. French, Mortimer Poncz

Blood (2017) 129 (26): 3486–3494.

https://doi.org/10.1182/blood-2017-02-770958

Published: 2017

Includes: Supplemental data

Journal Articles

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Blood (2016) 128 (22): 3.

https://doi.org/10.1182/blood.V128.22.3.3

Published: 2016

Journal Articles

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

Randolph B Lyde, Jr., BS, Hyunsook Ahn, Karen K Vo, BS, Danuta Jadwiga Jarocha, PhD, Li Zhai, BS, Spencer K. Sullivan, MD, Valder R Arruda, MD PhD, Denise E. Sabatino, PhD, Rodney M. Camire, PhD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2016) 128 (22): 2559.

https://doi.org/10.1182/blood.V128.22.2559.2559

Published: 2016

Journal Articles

Fli1 Expression Level Affects Megakaryopoiesis and Thrombopoiesis: More Is Not Always a Bad Thing

Karen K Vo, BS, Danuta Jadwiga Jarocha, PhD, Randolph B Lyde, Jr., BS, Spencer K. Sullivan, MD, Deborah French, PhD, Mortimer Poncz, MD

Blood (2016) 128 (22): 3876.

https://doi.org/10.1182/blood.V128.22.3876.3876

Published: 2016

Journal Articles

Comparative analysis of human ex vivo–generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale

Yuhuan Wang, Vincent Hayes, Danuta Jarocha, Xiuli Sim, Dawn C. Harper, Rudy Fuentes, Spencer K. Sullivan, Paul Gadue, Stella T. Chou, Beverly J. Torok-Storb, Michael S. Marks, Deborah L. French, Mortimer Poncz

Blood (2015) 125 (23): 3627–3636.

https://doi.org/10.1182/blood-2014-08-593053

Published: 2015

Includes: Supplemental data

Journal Articles

A(nother) day in the life of neonatal platelets

Spencer K. Sullivan, Mortimer Poncz

Blood (2014) 123 (22): 3372–3373.

https://doi.org/10.1182/blood-2014-03-562132

Published: 2014

Journal Articles

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia

Spencer K. Sullivan, Jason A. Mills, Sevasti B. Koukouritaki, Karen K. Vo, Randolph B. Lyde, Prasuna Paluru, Guoha Zhao, Li Zhai, Lisa M. Sullivan, Yuhuan Wang, Siddharth Kishore, Eyad Z. Gharaibeh, Michele P. Lambert, David A. Wilcox, Deborah L. French, Mortimer Poncz, Paul Gadue

Blood (2014) 123 (5): 753–757.

https://doi.org/10.1182/blood-2013-10-530725

Published: 2014

Includes: Supplemental data

Journal Articles

Clonal genetic and hematopoietic heterogeneity among human-induced pluripotent stem cell lines

Blood (2013) 122 (12): 2047–2051.

https://doi.org/10.1182/blood-2013-02-484444

Published: 2013

Includes: Supplemental data

Journal Articles

Patient-derived induced pluripotent stem cells recapitulate hematopoietic abnormalities of juvenile myelomonocytic leukemia

Shilpa Gandre-Babbe, Prasuna Paluru, Chiaka Aribeana, Stella T. Chou, Silvia Bresolin, Lin Lu, Spencer K. Sullivan, Sarah K. Tasian, Julie Weng, Helene Favre, John K. Choi, Deborah L. French, Mignon L. Loh, Mitchell J. Weiss

Blood (2013) 121 (24): 4925–4929.

https://doi.org/10.1182/blood-2013-01-478412

Published: 2013

Includes: Supplemental data

Journal Articles

Challenges and promises for the development of donor-independent platelet transfusions

Michele P. Lambert, Spencer K. Sullivan, Rudy Fuentes, Deborah L. French, Mortimer Poncz

Blood (2013) 121 (17): 3319–3324.

https://doi.org/10.1182/blood-2012-09-455428

Published: 2013

Journal Articles

Tissue-Specific Transgene Expression in Induced Pluripotent Stem (iPS) Cell-Derived Megakaryocytes: Correction of Glanzmann Thrombasthenia (GT)

Spencer K. Sullivan, MD, Jason A. Mills, PhD, Li Zhai, MD, Prasuna Paluru, MS, Karen Vo, Michele Lambert, MD, Sevasti B. Koukouritaki, PhD, Juan Fang, MD, David A. Wilcox, PhD, Deborah L. French, PhD, Mortimer Poncz, MD, Paul Gadue, PhD

Blood (2012) 120 (21): 387.

https://doi.org/10.1182/blood.V120.21.387.387

Published: 2012

Journal Articles

Multi-Center Phase II Study of Perifosine (KRX-0401) Alone and in Combination with Dexamethasone (dex) for Patients with Relapsed or Relapsed/Refractory Multiple Myeloma (MM): Promising Activity as Combination Therapy with Manageable Toxicity.

Blood (2007) 110 (11): 1164.

https://doi.org/10.1182/blood.V110.11.1164.1164

Published: 2007

Journal Articles

A Multicenter Phase II Study of Perifosine (KRX-0401) Alone and in Combination with Dexamethasone (Dex) for Patients with Relapsed or Relapsed/Refractory Multiple Myeloma (MM).

Blood (2006) 108 (11): 3582.

https://doi.org/10.1182/blood.V108.11.3582.3582

Published: 2006

Journal Articles

Human Thymocytes Express CCR-3 and Are Activated by Eotaxin

Karin Franz-Bacon, Daniel J. Dairaghi, Stefen A. Boehme, Susan K. Sullivan, Thomas J. Schall, Paul J. Conlon, Naomi Taylor, Kevin B. Bacon

Blood (1999) 93 (10): 3233–3240.

https://doi.org/10.1182/blood.V93.10.3233.410k33_3233_3240

Published: 1999

NARROW

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

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

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

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

FLI1 level during megakaryopoiesis affects thrombopoiesis and platelet biology

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression

Infusion of Coagulation Factor VIII-Containing Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Shows Potential As Hemophilia a Treatment

Fli1 Expression Level Affects Megakaryopoiesis and Thrombopoiesis: More Is Not Always a Bad Thing

Comparative analysis of human ex vivo–generated platelets vs megakaryocyte-generated platelets in mice: a cautionary tale

A(nother) day in the life of neonatal platelets

High-level transgene expression in induced pluripotent stem cell–derived megakaryocytes: correction of Glanzmann thrombasthenia

Clonal genetic and hematopoietic heterogeneity among human-induced pluripotent stem cell lines

Patient-derived induced pluripotent stem cells recapitulate hematopoietic abnormalities of juvenile myelomonocytic leukemia

Challenges and promises for the development of donor-independent platelet transfusions

Tissue-Specific Transgene Expression in Induced Pluripotent Stem (iPS) Cell-Derived Megakaryocytes: Correction of Glanzmann Thrombasthenia (GT)

Multi-Center Phase II Study of Perifosine (KRX-0401) Alone and in Combination with Dexamethasone (dex) for Patients with Relapsed or Relapsed/Refractory Multiple Myeloma (MM): Promising Activity as Combination Therapy with Manageable Toxicity.

A Multicenter Phase II Study of Perifosine (KRX-0401) Alone and in Combination with Dexamethasone (Dex) for Patients with Relapsed or Relapsed/Refractory Multiple Myeloma (MM).

Human Thymocytes Express CCR-3 and Are Activated by Eotaxin

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American Society of Hematology

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ASH Publications

American Society of Hematology

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