Author

Brett E. Crawford

BioMarin (United States)ORCID 0000-0002-7811-4564Unclaimed ORCID profile · follow to get notified of new preprints

Biology Disciplines

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Signature Assays, Organisms & Methods

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Approach:experimental assay· 2 Research Focus:cell type annotation· 1 Research Focus:drug response / drug discovery· 1 Organism:human· 1 Assay:proteomics· 1 Organism:rat· 1

Frequent Co-Authors

Amanda R. Luu· 1 shared Annemieke M Aartsma-RusiD· 1 shared Brian Heglar· 1 shared Bryan K. Yip· 1 shared C. Tanganyika-de WinteriD· 1 shared Chad Koehler· 1 shared Donnie Mackenzie· 1 shared Elizabeth M. van der PijliD· 1 shared

Activity by Year

2019-2026 · peak 1/yr

Co-Author Collaboration Network

10 top collaborators · 24 mutual co-author links

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1 shared paperGenetics

Amanda R. Luu

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2 papers

Recent·Most cited

Sequence determinants of efficient exon 44 skipping in Duchenne muscular dystrophy define design principles for steric-blocking antisense oligonucleotides

Enyang Han, Katherine Webster, Tiberiu Loredan Stan, C. Tanganyika-de Winter, Elizabeth M. van der Pijl, Jordan Tahquechi, Brian Heglar, Chad Koehler, Irinna Papangeli, Donnie Mackenzie, Brett E. Crawford, Annemieke M Aartsma-Rus, Tom A. Hartl

The paper identifies sequence and chemical features of ASOs that efficiently induce exon 44 skipping in DMD and defines design principles for therapeutic ASOs.

bioRxiv · Jul 1, 2026 · PreprintMolecular Biology

BMN 250, a fusion of lysosomal alpha-N-acetylglucosaminidase with IGF2, exhibits different patterns of cellular uptake into critical cell types of Sanfilippo syndrome B disease pathogenesis.

Gouri Yogalingam, Amanda R. Luu, Heather Prill, Melanie J. Lo, Bryan K. Yip, John Holtzinger, Terri Christianson, Mika Aoyagi-Scharber, Roger Lawrence, Brett E. Crawford, Jonathan H. LeBowitz

This study characterizes two distinct cellular uptake mechanisms for BMN 250 in Sanfilippo B disease, showing CI-MPR-mediated uptake in neurons and astrocytes and receptor-independent uptake in microglia.

PLoS ONE · Jan 18, 2019Peer-reviewedGenetics