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Journal Articles

Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

Maxwell Chappell, Laura Breda, Danuta Jadwiga Jarocha, Michael Triebwasser, Tyler E Papp, Valentina Ghiaccio, Megan T Fedorky, Amaliris Guerra, Kandace Gollomp, Osheiza Y. Abdulmalik, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Hamideh Parhiz, Stefano Rivella

Blood (2022) 140 (Supplement 1): 300–302.

https://doi.org/10.1182/blood-2022-166621

Published: 2022

Journal Articles

A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

Maxwell Chappell, Danuta Jadwiga Jarocha, Laura Breda, Valentina Ghiaccio, Michael Triebwasser, Megan Fedorky, Amaliris Guerra, Kandace Gollomp, Nattiya Teawtrakul, Stavros Glentis, Antonis Kattamis, Osheiza Abdulmalik, Stefano Rivella

Blood (2021) 138 (Supplement 1): 2012.

https://doi.org/10.1182/blood-2021-151217

Published: 2021

Journal Articles

Improved Gene Therapy for Metachromatic Leukodystrophy

Lucas Tricoli, Adeline Vanderver, Laura Adang, Maxwell Chappell, Laura Breda, Naoto Tanaka, Michael Triebwasser, David Wenger, Danuta Jadwiga Jarocha, Amaliris Guerra, Lars Schlotawa, Karthikeyan Radhakrishnan, Rebecca Ahrens-Nicklas, Peter Kurre, Stephanie Hurwitz, Stefano Rivella

Blood (2021) 138 (Supplement 1): 3979.

https://doi.org/10.1182/blood-2021-150986

Published: 2021

Journal Articles

Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

Michael Triebwasser, Danuta Jadwiga Jarocha, Laura Breda, Megan Fedorky, Stefano Rivella

Blood (2021) 138 (Supplement 1): 3131.

https://doi.org/10.1182/blood-2021-151040

Published: 2021

Journal Articles

In Situ Microscopy Studies of Infused Megakaryocytes: Implications in Thrombopoiesis

Hyunjun Kim, Danuta Jadwiga Jarocha, Ian Johnston, Hyunsook Ahn, Deborah L French, Lubica Rauova, Mortimer Poncz

Blood (2021) 138 (Supplement 1): 4287.

https://doi.org/10.1182/blood-2021-150764

Published: 2021

Journal Articles

The Inherited Platelet Disorder of RUNX1 Haploinsufficiency (Familial Platelet Disorder with Associated Myeloid Malignancy (FPDMM)) Has a Marked Deficiency of Megakaryocyte-Biased Hematopoietic Progenitor Cells: Mechanistic Studies and Drug Correction

Brian Estevez, PhD, Sara Borst, BS, Danuta Jadwiga Jarocha, PhD, Varun Sudunagunta, Michael Gonzalez, PhD, Peng Gao, Kai Tan, Deborah L French, PhD, Paul Gadue, PhD, Mortimer Poncz, MD

Blood (2019) 134 (Supplement_1): 220.

https://doi.org/10.1182/blood-2019-124851

Published: 2019

Journal Articles

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Laura Breda, PhD, Valentina Ghiaccio, PhD, Silvia Pires Lourenco, MSc, Danuta Jadwiga Jarocha, PhD, Yasuhiro Ikawa, MD PhD, Naoto Tanaka, PhD, Virginia Guzikowski, Carla Casu, PhD, Kim Smith-Whitley, MD, Janet L. Kwiatkowski, MD MSCE, Stefano Rivella, PhD

Blood (2019) 134 (Supplement_1): 2242.

https://doi.org/10.1182/blood-2019-124043

Published: 2019

Journal Articles

Janus Kinase (Jak) 1 Inhibition Affects Both Megakaryopoiesis and Thrombopoiesis

Danuta Jadwiga Jarocha, PhD, Paul Gadue, PhD, Wei Tong, PhD, Robert C Newton, PhD, Mortimer Poncz, MD

Blood (2018) 132 (Supplement 1): 2559.

https://doi.org/10.1182/blood-2018-99-115407

Published: 2018

Journal Articles

Enhancing Functional Platelet Release In Vivo from in Vitro-Grown Megakaryocytes Using the Protein Kinase Inhibitor SU6656

Danuta Jadwiga Jarocha, PhD, Karen K Vo, BS, Randolph B Lyde, Jr., BS, Vincent M Hayes, BS, Mortimer Poncz

Blood (2016) 128 (22): 1029.

https://doi.org/10.1182/blood.V128.22.1029.1029

Published: 2016

Journal Articles

Platelet Release from Infused CD34 + -Derived Human Megakaryocytes: Lessons from a Natural-Occurring Platelet Bioreactor

Ian Johnston, BS, Vincent M Hayes, BS, Jing Dai, MD PhD, Danuta Jadwiga Jarocha, PhD, Paul Kubes, PhD, Mortimer Poncz, MD, Lubica Rauova, MD PhD

Blood (2016) 128 (22): 1375.

https://doi.org/10.1182/blood.V128.22.1375.1375

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

The Influence of FLI1 Expression Levels on Megakaryopoiesis: Studies Using iPSCs

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

Blood (2015) 126 (23): 2352.

https://doi.org/10.1182/blood.V126.23.2352.2352

Published: 2015

Journal Articles

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

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

Blood (2015) 126 (23): 2266.

https://doi.org/10.1182/blood.V126.23.2266.2266

Published: 2015

NARROW

Lnp-Targeting Hematopoietic Stem Cells and Lentiviral Gene Transfer to Generate and Rescue a Novel Mouse Model of Lethal Alpha-Thalassemia

A Severe Mouse Model of Alpha-Thalassemia to Study Abnormal Iron Metabolism and Erythropoiesis, Hematopoietic Stem Cell Behavior and Development of a Gene Therapy Approach for Its Treatment

Improved Gene Therapy for Metachromatic Leukodystrophy

Rescue of Murine IL-7 Receptor Deficiency with Human IL-7 Receptor Gene Therapy

In Situ Microscopy Studies of Infused Megakaryocytes: Implications in Thrombopoiesis

The Inherited Platelet Disorder of RUNX1 Haploinsufficiency (Familial Platelet Disorder with Associated Myeloid Malignancy (FPDMM)) Has a Marked Deficiency of Megakaryocyte-Biased Hematopoietic Progenitor Cells: Mechanistic Studies and Drug Correction

Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies

Janus Kinase (Jak) 1 Inhibition Affects Both Megakaryopoiesis and Thrombopoiesis

Enhancing Functional Platelet Release In Vivo from in Vitro-Grown Megakaryocytes Using the Protein Kinase Inhibitor SU6656

Platelet Release from Infused CD34 + -Derived Human Megakaryocytes: Lessons from a Natural-Occurring Platelet Bioreactor

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

The Influence of FLI1 Expression Levels on Megakaryopoiesis: Studies Using iPSCs

Towards the Care of Hemophilia a Patients Using Induced Pluripotent Stem Cell (iPSC)-Derived Megakaryocytes (iMks) Expressing Coagulation Factor (F) VIII

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American Society of Hematology

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American Society of Hematology

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