Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease

Margarita M. Ivanova; Julia Dao; Neil Kasaci; Benjamin Adewale; Shaista Nazari; Lauren Noll; Jacqueline Fikry; Armaghan Hafez Sanati; Ozlem Goker-Alpan

doi:10.1371/journal.pone.0247211

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Open Access

Peer-reviewed

Research Article

Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease

Margarita M. Ivanova ,

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

* E-mail: mivanova@ldrtc.org

¶‡ MMI and OGA also contributed equally to this work.

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America

https://orcid.org/0000-0002-6197-8606

⨯
Julia Dao,

Roles Formal analysis, Investigation, Visualization, Writing – original draft

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Neil Kasaci,

Roles Investigation, Visualization

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Benjamin Adewale,

Roles Investigation, Visualization

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Shaista Nazari,

Roles Investigation

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Lauren Noll ,

Contributed equally to this work with: Lauren Noll, Jacqueline Fikry

Roles Investigation, Resources

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Jacqueline Fikry ,

Contributed equally to this work with: Lauren Noll, Jacqueline Fikry

Roles Investigation, Resources

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Armaghan Hafez Sanati,

Roles Investigation

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯
Ozlem Goker-Alpan

Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Methodology, Project administration, Resources, Supervision, Writing – review & editing

¶‡ MMI and OGA also contributed equally to this work.

Affiliation Lysosomal and Rare Disorders Research and Treatment Center, Fairfax, VA, United States of America
⨯

Cellular and biochemical response to chaperone versus substrate reduction therapies in neuropathic Gaucher disease

Margarita M. Ivanova,
Julia Dao,
Neil Kasaci,
Benjamin Adewale,
Shaista Nazari,
Lauren Noll,
Jacqueline Fikry,
Armaghan Hafez Sanati,
Ozlem Goker-Alpan

Published: October 25, 2021
https://doi.org/10.1371/journal.pone.0247211

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