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Nature Communications|June 30, 2019
CRISPR-Cas9 fusion to dominant-negative 53BP1 enhances HDR and inhibits NHEJ specifically at Cas9 target sitesRajeswari Jayavaradhan, Devin M Pillis, Michael Goodman, et al.
Plos One|October 3, 2012
Stem cell selection in vivo using foamy vectors cures canine pyruvate kinase deficiencyGrant D Trobridge, Brian C Beard, Robert A Wu, et al.
British Journal of Haematology|March 9, 2025
Real-world insights from a cohort of approximately 2000 individuals who were analysed using a freely available next-generation sequencing anaemia screening programmeJorune Balciuniene, Saliha Yilmaz, Satheesh Chonat, et al.
Human Gene Therapy|July 30, 2016
High Level of Perforin Expression Is Required for Effective Correction of Hemophagocytic LymphohistiocytosisSwati Tiwari, Adrianne Hontz, Catherine E Terrell, et al.
Molecular and Cellular Biology|November 19, 2014
MicroRNA 648 Targets ET-1 mRNA and is cotranscriptionally regulated with MICAL3 by PAX5Chen Li, Caryn S Gonsalves, Marthe-Sandrine Eiymo Mwa Mpollo, et al.
Angiogenesis|April 8, 2022
NRASQ61R mutation in human endothelial cells causes vascular malformationsElisa Boscolo, Patricia Pastura, Sandra Schrenk, et al.
The Journal of Biological Chemistry|November 13, 2014
Peroxisome proliferator-activated receptor-α-mediated transcription of miR-199a2 attenuates endothelin-1 expression via hypoxia-inducible factor-1αChen Li, Marthe-Sandrine Eiymo Mwa Mpollo, Caryn S Gonsalves, et al.
Blood Advances|May 4, 2021
FT-4202, an oral PKR activator, has potent antisickling effects and improves RBC survival and Hb levels in SCA miceArchana Shrestha, Mengna Chi, Kimberly Wagner, et al.
Blood|April 5, 2008
Efficient transduction of pigtailed macaque hematopoietic repopulating cells with HIV-based lentiviral vectorsGrant D Trobridge, Brian C Beard, Christina Gooch, et al.
American Journal of Respiratory and Critical Care Medicine|November 28, 2013
Use of induced pluripotent stem cells to recapitulate pulmonary alveolar proteinosis pathogenesisTakuji Suzuki, Christopher Mayhew, Anthony Sallese, et al.
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