SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis-Reference-Cited by-同舟云学术

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SP-101, A Novel Adeno-Associated Virus Gene Therapy for the Treatment of Cystic Fibrosis, Mediates Functional Correction of Primary Human Airway Epithelia From Donors with Cystic Fibrosis

Published:2024-08-29 Issue: Volume: Page:
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Excoffon Katherine JDA¹^ORCID,Lin Shen¹,Narayan Poornima Kotha Lakshmi¹,Sitaraman Sneha¹,Jimah Awal M.¹,Fallon Tyler T.¹,James Melane L.¹,Glatfelter Matthew R.¹,Limberis Maria P.¹^ORCID,Smith Mark D.¹,Guffanti Guia²,Kolbeck Roland¹

Affiliation:

1. Spirovant Sciences, Inc., Philadelphia, Pennsylvania, USA

2. Sumitomo Pharma America, New York, New York, USA.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2024.063

Reference37 articles.

1. CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes

2. Lentiviral-mediated phenotypic correction of cystic fibrosis pigs

3. Progress in Respiratory Gene Therapy

4. Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward

5. AAV vectors: The Rubik’s cube of human gene therapy

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