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Nirav Malani
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Journal Articles

Immune Reconstitution and Preliminary Safety Analysis Of 9 Patients Treated With Somatic Gene Therapy For X-Linked Severe Combined Immunodeficiency (SCID-X1) With a Self-Inactivating Gammaretroviral Vector

Salima Hacein-Bey-Abina, Pharm.D., Ph.D., Sung-Yun Pai, MD, Frederic Bushman, PhD, Myriam Armant, PhD, Stephane Blanche, MD, Johanna Blondeau, Laure Caccavelli, Heather Daley, BS, Colleen Dansereau, RN, BSN, CPN, CPHON, Satiro deOliveira, MD, Dongjing Guo, MPH, Gregory Hopkins, BS, Annick Lim, M.Sci, Wendy B. London, PHD, Nirav Malani, Frances Male, BA, Punam Malik, MD, Maria Angelica Marinovic, MD, Sean McDonough, MS, Matias Oleastro, MD, Capucine Picard, MD PhD, Jerome Ritz, MD, Axel Schambach, MD PhD, Leslie E Silberstein, MD, Alla Tsytsykova, PhD, Johannes C.M. Van der Loo, PhD, Christopher Baum, MD, H. Bobby Gaspar, MD PhD, Donald B. Kohn, MD, Alexandra H. Filipovich, MD, Luigi Daniele Notarangelo, MD, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2013) 122 (21): 715.
https://doi.org/10.1182/blood.V122.21.715.715
Published: 2013
Journal Articles

Assessing the potential for AAV vector genotoxicity in a murine model

Hojun Li, Nirav Malani, Shari R. Hamilton, Alexander Schlachterman, Giulio Bussadori, Shyrie E. Edmonson, Rachel Shah, Valder R. Arruda, Federico Mingozzi, J. Fraser Wright, Frederic D. Bushman, Katherine A. High
Journal: Blood
Blood (2011) 117 (12): 3311–3319.
https://doi.org/10.1182/blood-2010-08-302729
Published: 2011
Includes: Supplemental data
Journal Articles

The host genomic environment of the provirus determines the abundance of HTLV-1–infected T-cell clones

Nicolas A. Gillet, Nirav Malani, Anat Melamed, Niall Gormley, Richard Carter, David Bentley, Charles Berry, Frederic D. Bushman, Graham P. Taylor, Charles R. M. Bangham
Journal: Blood
Blood (2011) 117 (11): 3113–3122.
https://doi.org/10.1182/blood-2010-10-312926
Published: 2011
Includes: Supplemental data
Journal Articles

Therapeutic Transgene Expression From Genomic Safe Harbors In Patient-Specific Induced Pluripotent Stem Cells

Eirini P Papapetrou, MD, PhD, Gabsang Lee, PhD, Nirav Malani, Manu Setty, Isabelle Riviere, PhD, Laxmi MS Tirunagari, Kyuichi Kadota, MD, Shoshannah L Roth, PhD, Patricia Giardina, MD, Agnes Viale, PhD, Christina Leslie, PhD, Frederic Bushman, PhD, Lorenz Studer, MD, Michel Sadelain, MD, PhD
Journal: Blood
Blood (2010) 116 (21): 564.
https://doi.org/10.1182/blood.V116.21.564.564
Published: 2010
Journal Articles

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

Gary P. Wang, Charles C. Berry, Nirav Malani, Philippe Leboulch, Alain Fischer, Salima Hacein-Bey-Abina, Marina Cavazzana-Calvo, Frederic D. Bushman
Journal: Blood
Blood (2010) 115 (22): 4356–4366.
https://doi.org/10.1182/blood-2009-12-257352
Published: 2010
Includes: Supplemental data
Journal Articles

Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice.

Hojun Li, Nirav Malani, Shari R Hamilton, DVM, Alexander Schlachterman, MD, Jim Z Zhang, PhD, Giulio Bussadori, Shyrie A Edmonson, Federico Mingozzi, PhD, J. Fraser Wright, PhD, Valder R. Arruda, MD, PhD, Frederic D Bushman, PhD, Katherine A. High, MD
Journal: Blood
Blood (2009) 114 (22): 2465.
https://doi.org/10.1182/blood.V114.22.2465.2465
Published: 2009