Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease-Reference-Cited by-同舟云学术

Ionizable Lipid Nanoparticles for Therapeutic Base Editing of Congenital Brain Disease

Published:2023-07-17 Issue:14 Volume:17 Page:13594-13610
ISSN:1936-0851
Container-title:ACS Nano
language:en
Short-container-title:ACS Nano

Author:

Palanki Rohan¹²^ORCID,Bose Sourav K.²,Dave Apeksha²,White Brandon M.²,Berkowitz Cara²,Luks Valerie²,Yaqoob Fazeela³,Han Emily¹,Swingle Kelsey L.¹^ORCID,Menon Pallavi²,Hodgson Emily²,Biswas Arijit⁴,Billingsley Margaret M.¹^ORCID,Li Li⁵,Yiping Fan⁴,Carpenter Marco²,Trokhan Alexandra²,Yeo Julie⁴,Johana Nuryanti⁴,Wan Tan Yi⁴,Alameh Mohamad-Gabriel⁶^ORCID,Bennett Frederick Chris³,Storm Phillip B.⁷,Jain Rajan⁶⁵,Chan Jerry⁴⁸,Weissman Drew⁶^ORCID,Mitchell Michael J.¹⁹¹⁰⁵¹¹^ORCID,Peranteau William H.²¹²

Affiliation:

1. Department of Bioengineering, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

2. Center for Fetal Research, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States

3. Department of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

4. Duke-NUS Graduate Medical School, Singapore, 169547, Singapore

5. Cardiovascular Institute, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

6. Department of Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

7. Division of Neurosurgery, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States

8. Department of Reproductive Medicine, KK Women’s and Children’s Hospital, Singapore, 229899, Singapore

9. Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

10. Institute for Immunology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania 19104, United States

11. Institute for Regenerative Medicine, Perelman School of Medicine, Philadelphia, Pennsylvania 19104, United States

12. Division of General, Thoracic, and Fetal Surgery, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania 19104, United States

Funder

NIH Office of the Director

National Institute of Diabetes and Digestive and Kidney Diseases

National Heart, Lung, and Blood Institute

Burroughs Wellcome Fund

Publisher

American Chemical Society (ACS)

Subject

General Physics and Astronomy,General Engineering,General Materials Science

Link

https://pubs.acs.org/doi/pdf/10.1021/acsnano.3c02268

Reference47 articles.

1. Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

2. Neurology of inherited glycosylation disorders

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4. Delivery technologies for in utero gene therapy

5. Therapeutic genome editing: prospects and challenges

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