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William Querbes
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Journal Articles

In Vivo RNA delivery by targeted lipid nanoparticles enable gene editing in hematopoietic stem cells and T cells

Free
Rahul Palchaudhuri, Michael Monte, Mohit Gupta, Giulia Schiroli, Lorenzo Tozzi, Lei Liu, Han Gu, Rebecca Goldman, Apiwat Wangweerawong, Aaron Zannini, Yaser Heshmati, Christina Ferren, Alyssa Sanfilippo, Tiffany Morales, Jessica Desmond, Kartika Venugopal, Jason Rodriguez, Albert De Iaco, Cecilia Cotta-Ramusino, William E. Salomon, James Rottman, William Querbes, Hari Pujar, Michael C Holmes, Z. Jane Wang
Journal: Blood
Blood (2025) 146 (Supplement 1): 4318.
https://doi.org/10.1182/blood-2025-4318
Published: 2025
Journal Articles

Targeted lnp delivery of an RNA gene writer In Vivo enables generation of functional CAR-T cells

Free
Cecilia Cotta-Ramusino, Albert De Iaco, James Rottman, Jason Rodriguez, Donghui Li, Kartika Venugopal, Jessica Desmond, Christina Ferren, Mohit Gupta, Rebecca Levy, Rahul Palchaudhuri, Z. Jane Wang, William Querbes, Hari Pujar, Michael C Holmes
Journal: Blood
Blood (2025) 146 (Supplement 1): 6097.
https://doi.org/10.1182/blood-2025-6097
Published: 2025
Journal Articles

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

Free
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

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

Free
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

Interim Data from a Randomized, Placebo Controlled, Phase 1 Study of Aln-AS1, an Investigational RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria

Free
Eliane Sardh, MD PhD, Pauline Harper, MD PhD, Nabil Al-Tawil, MD, Manisha Balwani, MD MS, Karl Anderson, MD, Joseph Bloomer, MD, D. Montgomery Bissel, MD, Robert J. Desnick, MD, Charles J. Parker, MD, John D. Phillips, PhD, Herbert L. Bonkovsky, MD, Craig Penz, MA, Amy Chan, PhD, Chang-Heok Soh, PhD, William Querbes, PhD, Amy Simon, MD, Penelope Stein, MD PhD, David C. Rees, FRCP, FRCPath, MA, MBBS
Journal: Blood
Blood (2016) 128 (22): 2318.
https://doi.org/10.1182/blood.V128.22.2318.2318
Published: 2016
Journal Articles

Treatment of erythropoietin deficiency in mice with systemically administered siRNA

Free
William Querbes, Roman L. Bogorad, Javid Moslehi, Jamie Wong, Amy Y. Chan, Elena Bulgakova, Satya Kuchimanchi, Akin Akinc, Kevin Fitzgerald, Victor Koteliansky, William G. Kaelin, Jr
Journal: Blood
Blood (2012) 120 (9): 1916–1922.
https://doi.org/10.1182/blood-2012-04-423715
Published: 2012
Includes: Supplemental data
Journal Articles

Liver Specific Delivery of siRNA Targeting EGLN Prolyl Hydroxylases Activates Hepatic Erythropoietin Production and Stimulates Erythropoiesis,

Free
William Querbes, PhD, Roman Bogorad, PhD, Javid Moslehi, MD, Akin Akinc, PhD, Jamie Wong, PhD, Chris Zurenko, June Qin, Julia Hettinger, Satya Kuchimanchi, PhD, Klaus Charisse, PhD, Dinah WY Sah, PhD, Kevin Fitzgerald, PhD, Victor Kotelianski, MD, PhD, William G. Kaelin, Jr., MD
Journal: Blood
Blood (2011) 118 (21): 3161.
https://doi.org/10.1182/blood.V118.21.3161.3161
Published: 2011