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Journal Articles

In Vivo HSC Gene Editing for Correction of the Sickle Cell Mutation Using RNA Gene Writers

Lorenzo Tozzi, Giulia Schiroli, Yaser Heshmati, Yu Cao, Michael T. Monte, Apiwat Wangweerawong, James B. Rottman, Rahul Palchaudhuri, Jean-Antoine Ribeil, John Manis, Hari Pujar, William Querbes, Anne Bothmer, Cecilia Cotta-Ramusino, Z. Jane Wang, William E. Salomon, Michael C Holmes
Journal: Blood
Blood (2024) 144 (Supplement 1): 515.
https://doi.org/10.1182/blood-2024-208626
Published: 2024
Journal Articles

Targeted Lipid Nanoparticle Delivery of an RNA Gene Writer In Vivo Enables Generation of CAR-T Cells in a Humanized Mouse Model

Jason Rodriguez, Alberto De Iaco, James B. Rottman, Donghui Liu, Kartika Venugopal, Xin Qiao, Yu Cao, Rebecca Levy, Mohit Gupta, Rahul Palchaudhuri, Bill Querbes, Cecilia Cotta-Ramusino, Hari Pujar, Madhusudan V Peshwa, Michael C Holmes
Journal: Blood
Blood (2024) 144 (Supplement 1): 4799.
https://doi.org/10.1182/blood-2024-208176
Published: 2024
Journal Articles

Delivery of Gene Writers In Vivo to Hematopoietic Stem Cells and T Cells Using Targeted and Untargeted Lipid Nanoparticles (LNP)

Michael T. Monte, Lorenzo Tozzi, Rahul Palchaudhuri, Apiwat Wangweerawong, Yaser Heshmati, Giulia Schiroli, Han Gu, Yu Cao, William E. Salomon, Anne Bothmer, Jason Rodriguez, Mohit Gupta, James B. Rottman, William Querbes, Cecilia Cotta-Ramusino, Michael C Holmes, Hari Pujar, Z. Jane Wang
Journal: Blood
Blood (2024) 144 (Supplement 1): 2197.
https://doi.org/10.1182/blood-2024-208968
Published: 2024
Journal Articles

Alloengraftment without significant toxicity or GVHD in CD45 antibody-drug conjugate–conditioned Fanconi anemia mice

Asim Saha, Rahul Palchaudhuri, Leanne Lanieri, Sharon Hyzy, Megan J. Riddle, Jamie Panthera, Cindy R. Eide, Jakub Tolar, Angela Panoskaltsis-Mortari, Lev Gorfinkel, Victor Tkachev, Ulrike Gerdemann, Francesca Alvarez-Calderon, Elisa Rojas Palato, Margaret L. MacMillan, John E. Wagner, Leslie S. Kean, Mark J. Osborn, Hans-Peter Kiem, David T. Scadden, Lisa M. Olson, Bruce R. Blazar
Journal: Blood
Blood (2024) 143 (21): 2201–2216.
https://doi.org/10.1182/blood.2023023549
Published: 2024
Includes: Supplemental data
Journal Articles

In Vivo Delivery of RNA By Lipid Nanoparticles (LNP) to Hematopoietic Stem Cells and T Cells in Humanized Mice and Non-Human Primates

Michael T. Monte, Han Gu, Laura Saunders, Mohit Gupta, Lei Liu, Apiwat Wangweerawong, Thomas V. Colace, Aaron Zannini, Lorenzo Tozzi, Yu Cao, Giulia Schiroli, William E. Salomon, James B. Rottman, Rahul Palchaudhuri, Ulrike Gerdemann, Lev Gorfinkel, Francesca Alvarez Calderon, Leslie Kean, Hari Pujar, Z. Jane Wang
Journal: Blood
Blood (2023) 142 (Supplement 1): 4818.
https://doi.org/10.1182/blood-2023-187271
Published: 2023
Journal Articles

CD117 Antibody-Drug Conjugate Conditioning Allows Efficient Engraftment of Gene-Modified CD34+ Cells with Fertility Preservation in a Rhesus Gene Therapy Model

Naoya Uchida, Selami Demirci, Anh Le, Rebecca Chu, Xiong Liu, Ling Su, Xiaolin Wu, Allen Krouse, N Seth Linde, Aylin Bonifacino, So Gun Hong, Cynthia E. Dunbar, Leanne Lanieri, Anjali Bhat, Rahul Palchaudhuri, Bindu Bennet, Megan D. Hoban, Kirk Bertelsen, Lisa Olson, Robert Donahue, John F. Tisdale
Journal: Blood
Blood (2023) 142 (Supplement 1): 486.
https://doi.org/10.1182/blood-2023-177815
Published: 2023
Journal Articles

The Impact of CD45-Antibody-Drug Conjugate Conditioning on Clonal Dynamics and Immune Tolerance Post HSPC Transplantation in Rhesus Macaques

Taha Bartu Hayal, Chuanfeng Wu, Diana Abraham, Selami Demirci, Rahul Palchaudhuri, Leanne Lanieri, Anjali Bhat, Tiffany Tate, Zhuyan Guo, So Gun Hong, Lisa Olson, Robert Donahue, John F. Tisdale, Cynthia E. Dunbar
Journal: Blood
Blood (2023) 142 (Supplement 1): 3419.
https://doi.org/10.1182/blood-2023-182066
Published: 2023
Journal Articles

The Pharmacokinetic and Pharmacodynamic Characterization of Mgta-117, an Anti-CD117-Amanitin Antibody-Drug Conjugate for Targeted Conditioning Prior to Transplant, in Nonhuman Primates

Kirk Bertelsen, Leanne Lanieri, Rahul Palchaudhuri, Lisa Olson
Journal: Blood
Blood (2022) 140 (Supplement 1): 10227–10228.
https://doi.org/10.1182/blood-2022-159712
Published: 2022
Journal Articles

A CD45-targeted antibody-drug conjugate successfully conditions for allogeneic hematopoietic stem cell transplantation in mice

Asim Saha, Sharon Hyzy, Tahirih Lamothe, Katelyn Hammond, Nicholas Clark, Leanne Lanieri, Prashant Bhattarai, Rahul Palchaudhuri, Geoffrey O. Gillard, Jennifer Proctor, Megan J. Riddle, Angela Panoskaltsis-Mortari, Margaret L. MacMillan, John E. Wagner, Hans-Peter Kiem, Lisa M. Olson, Bruce R. Blazar
Journal: Blood
Blood (2022) 139 (11): 1743–1759.
https://doi.org/10.1182/blood.2021012366
Published: 2022
Includes: Supplemental data
Journal Articles

CD117 Antibody Drug Conjugate-Based Conditioning Allows for Efficient Engraftment of Gene-Modified CD34+ Cells in a Rhesus Gene Therapy Model

Naoya Uchida, Ulana Stasula, Malikiya Hinds, Paula Germino-Watnick, Allen E. Krouse, N Seth Linde, Aylin C. Bonifacino, Kellie Latimer, Prashant Bhattarai, Nicholas C. Yoder, Rahul Palchaudhuri, Qing Li, Kirk Bertelsen, Lisa Olson, Robert E. Donahue, John F. Tisdale
Journal: Blood
Blood (2021) 138 (Supplement 1): 560.
https://doi.org/10.1182/blood-2021-145524
Published: 2021
Journal Articles

Single Agent CD117-Targeted Antibody Drug Conjugate in Combination with Lymphodepleting Antibodies Enables Allogenic Hematopoietic Stem Cell Transplantation in Mice without Chemotherapy or Radiation

Leanne Lanieri, Tahirih Lamothe, Prashant Bhattarai, Rahul Palchaudhuri, Lisa Olson
Journal: Blood
Blood (2021) 138 (Supplement 1): 1682.
https://doi.org/10.1182/blood-2021-147905
Published: 2021
Journal Articles

Single Agent CD45-Targeted Antibody Drug Conjugate Enables Full Mismatch Allogeneic Hematopoietic Stem Cell Transplantation in a Murine HSCT Model

Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Geoff O Gillard, PhD, Katelyn J. Hammond, BS, Ganapathy N. Sarma, PhD, Asim Saha, PhD, Nicholas Clark, PhD, Anjali Bhat, MS, Tahirih L Lamothe, BS, Rahul Palchaudhuri, PhD, Bradley R Pearse, PhD, Charlotte F McDonagh, PhD, Hans-Peter Kiem, MDPhD, John E Wagner, MD, Bruce R. Blazar, MD, Anthony E Boitano, PhD, Michael P Cooke, PhD, John C. Davis, Jr., MD MPH, MS
Journal: Blood
Blood (2020) 136 (Supplement 1): 31.
https://doi.org/10.1182/blood-2020-140489
Published: 2020
Journal Articles

Reversing Clonal Hematopoiesis and Associated Atherosclerotic Disease By Targeted Antibody-Drug-Conjugate (ADC) Conditioning and Transplant

Karin Gustafsson, PhD, Catherine S Rhee, PhD, Elizabeth W Scadden, Vanessa Frodermann, Rahul Palchaudhuri, PhD, Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Geoff O Gillard, PhD, Anthony E Boitano, PhD, Michael P. Cooke, PhD, Matthias Nahrendorf, PhD, David T. Scadden, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 34–35.
https://doi.org/10.1182/blood-2020-143176
Published: 2020
Journal Articles

A Single Dose of a Novel Anti-Human CD117-Amanitin Antibody Drug Conjugate (ADC) Engineered for a Short Half-Life Provides Dual Conditioning and Anti-Leukemic Activity and Extends Survival Compared to Standard of Care in Multiple Preclinical Models of Acute Myeloid Leukemia (AML)

Leanne Lanieri, Tahirih L Lamothe, BS, Oliver Miske, Sean M McDonough, Ganapathy N Sarma, Prashant Bhattarai, Kellie Latimer, Junia Dushime, Nidhi Jain, Rahul Palchaudhuri, PhD, Ryan Knihtila, Bradley R Pearse, PhD, Jennifer L Proctor, BS, Anthony E Boitano, PhD, Michael P Cooke, PhD, John C. Davis, Jr., MD MPH, MS
Journal: Blood
Blood (2020) 136 (Supplement 1): 47–48.
https://doi.org/10.1182/blood-2020-140422
Published: 2020
Journal Articles

A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates

John F. Tisdale, MD, Robert E. Donahue, VMD, Naoya Uchida, MD, Bradley R Pearse, PhD, Sean M McDonough, MS, Jennifer L Proctor, BS, Allen Krouse, Nathaniel S. Linde, Aylin C. Bonifacino, Rajiv Panwar, PhD, Ganapathy N. Sarma, PhD, Lena Kien, BS, Kellie Latimer, Junia Dushime, BS, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Rahul Palchaudhuri, PhD, Qing Li, PhD, Pranoti Sawant, MS, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 610.
https://doi.org/10.1182/blood-2019-125968
Published: 2019
Journal Articles

A Novel Targeted Approach to Achieve Immune System Reset: CD45-Targeted Antibody Drug Conjugates Enable Autologous HSCT, Ameliorate Disease in Autoimmune Models, Potently Kill Human Immune Cells from Normal Donors and MS Patients, and Achieve Immune Depletion in Non-Human Primates (NHP)

Geoff O Gillard, Jennifer L Proctor, BS, Melissa L. Brooks, BS, Tahirih L Lamothe, BS, Sharon L. Hyzy, MS, Oliver Mikse, Sean McDonough, Rahul Palchaudhuri, PhD, Anjali Bhat, MS, Ganapathy N. Sarma, PhD, Prashant Bhattarai, Pranoti Sawant, MS, Bradley R Pearse, PhD, Charlotte F. Mcdonough, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3208.
https://doi.org/10.1182/blood-2019-130752
Published: 2019
Journal Articles

Targeting CD45 with an Amanitin Antibody-Drug Conjugate Effectively Depletes Human HSCs and Immune Cells for Transplant Conditioning

Rahul Palchaudhuri, PhD, Bradley R Pearse, PhD, Jennifer L Proctor, BS, Sharon L. Hyzy, MS, Sharon Aslanian, MS, Sean M McDonough, MS, Ganapathy Sarma, PhD, Melissa L. Brooks, BS, Anjali Bhat, MS, Danielle Ladwig, MS, Molly A McShea, MS, Nathan M Kallen, BA, Qing Li, PhD, Rajiv Panwar, PhD, Junia Dushime, BS, Pranoti Sawant, MS, Hillary L Adams, MS, Patrick C. Falahee, PhD, Tahirih L Lamothe, Andrew D Gabros, Lena Kien, BS, Geoff Gillard, PhD, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 4526.
https://doi.org/10.1182/blood-2018-99-117167
Published: 2018
Journal Articles

Antibody Drug Conjugates Targeted to CD45 or CD117 Enable Allogeneic Hematopoietic Stem Cell Transplantation in Animal Models

Rahul Palchaudhuri, PhD, Sharon L. Hyzy, MS, Jennifer L Proctor, BS, Hillary L Adams, MS, Bradley R Pearse, PhD, Ganapathy Sarma, PhD, Sharon Aslanian, MS, Geoff Gillard, PhD, Tahirih L Lamothe, Olga Burenkova, BS, Melissa L. Brooks, BS, Andrew D Gabros, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3324.
https://doi.org/10.1182/blood-2018-99-119432
Published: 2018
Journal Articles

CD117-Amanitin Antibody Drug Conjugates Effectively Deplete Human and Non-Human Primate HSCs: Proof of Concept As a Targeted Strategy for Conditioning Patients for Bone Marrow Transplant

Bradley R Pearse, PhD, Sean M McDonough, MS, Jennifer L Proctor, BS, Rajiv Panwar, PhD, Ganapathy Sarma, PhD, Molly A McShea, MS, Lena Kien, BS, Junia Dushime, BS, Hillary L Adams, MS, Sharon L. Hyzy, MS, Melissa L. Brooks, BS, Rahul Palchaudhuri, PhD, Qing Li, PhD, Nathan M Kallen, BA, Pranoti Sawant, MS, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3314.
https://doi.org/10.1182/blood-2018-99-114881
Published: 2018
Journal Articles

Single Doses of Antibody Drug Conjugates (ADCs) Targeted to CD117 or CD45 Have Potent In Vivo Anti-Leukemia Activity and Survival Benefit in Patient Derived AML Models

Jennifer L Proctor, BS, Sharon L. Hyzy, MS, Hillary L Adams, MS, Melissa L. Brooks, BS, Andrew D Gabros, Sean M McDonough, MS, Lena Kien, BS, Sharon Aslanian, MS, Bradley R Pearse, PhD, Rahul Palchaudhuri, PhD, Qing Li, PhD, Nathan M Kallen, BA, Ganapathy Sarma, PhD, Molly A McShea, MS, Danielle Ladwig, MS, Junia Dushime, BS, Rajiv Panwar, PhD, Charlotte F McDonagh, PhD, Anthony E. Boitano, PhD, Michael P. Cooke, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 3316.
https://doi.org/10.1182/blood-2018-99-112726
Published: 2018