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Your search keyword '"Deepa Rajan"' showing total 4 results
Start Over "Deepa Rajan" Journal molecular genetics and metabolism
4 results on '"Deepa Rajan"'

1. Interim analysis of key clinical outcomes from a phase 1/2 study of weekly intravenous DNL310 (brain-penetrant enzyme replacement therapy) in MPS II

Author

Joseph Muenzer, Paul Harmatz, Barbara K. Burton, Deepa Rajan, Simon A. Jones, Steven D. Chessler, Natalie Engmann, Adam Scheller, Charlene Chen, Rupa Caprihan, Akhil Bhalla, Tony Hung, Jason Nachtigall, Imanol Zubizarreta, Angela Kay, Simona Costanzo, Jeffrey M. Harris, Yuda Zhu, Peter Chin, Matthew D. Troyer, Carole Ho, and Anna I. Bakardjiev

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2023
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2. Interim results of Transpher A, a multicenter, single-dose clinical trial of UX111 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)

Author

Kevin M. Flanigan, Nicholas Smith, Maria Luz Couce, Deepa Rajan, Kristen Truxal, Kim L. McBride, Maria Jose de Castro Lopez, Maria Fuller, John Taylor, Ana B. Del Campo, Igor Grachev, and Heather Lau

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2023
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4. Phase 1/2 study update of an AAV9-based gene therapy for Gaucher disease type 2 (PROVIDE trial)

Author

Sarah Neuhaus, Travis B. Lewis, Paul A. Tamburri, Chester B. Whitley, Deepa Rajan, Renata C. Gallagher, Simon Jones, Aimee Donald, Maria L. Escolar, Yael Beckerman, Erin Mahoney, Daniel A. Hatch, Lee Shaughnessy, Patricia Sondergaard, Olga Uspenskaya, and Jeffrey Sevigny

Subjects
Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
Published
2023
Full Text
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