Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease.

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Bibliographic Details
Title: Long-term stability of posttranscriptional genetic silencing of BCL11A using a shmiR vector in Sickle Cell Disease.
Authors: Esrick EB; Boston Children's Hospital, Boston, Massachusetts, United States., Lehmann L; Division of Pediatric Hematology/Oncology, Dana-Farber Cancer Institute, Boston, MA, United States., Federico A; Boston Children's Hospital, Boston, MA, Boston, Massachusetts, United States., Vincon H; Boston Children's Hospital, Boston, Massachusetts, United States., Liu B; Boston Children's Hospital, Boston, Massachusetts, United States., Daley H; Dana-Farber Cancer Institute, Boston, Massachusetts, United States., Dansereau C; Dana-Farber Cancer Institute, Boston, Massachusetts, United States., De Oliveira S; University of California, Los Angeles, Los Angeles, California, United States., Everett JK; University of Pennsylvania, Philadelphia, Pennsylvania, United States., Kao PC; Boston Children's Hospital/Dana-Farber Cancer Institute, Boston, Massachusetts, United States., Moore TB; UCLA, Los Angeles, CA, California, United States., Morris E; Dana-Farber Cancer Institute, Boston, Massachusetts, United States., Trebeden-Negre H; CellforCure, Les Ulis, France., Shaw KL; Boston Children's Hospital, Boston, Massachusetts, United States., Roach GD; Seattle Children's Hospital, Seattle, Washington, United States., Ritz J; Dana-Farber Cancer Institute; Harvard Medical School, Boston, Massachusetts, United States., Roche AM; University of Pennsylvania, Philadelphia, Pennsylvania, United States., Silva O; Boston Children's Hospital, Boston, Massachusetts, United States., Grant PE; Boston Children's Hospital, Boston, Massachusetts, United States., Bushman FD; University of Pennsylvania, Philadelphia, Pennsylvania, United States., Kohn DB; University of California, Los Angeles, Los Angeles, California, United States., Jain A; Loma Linda University, Redlands, California, United States., London WB; Boston Children's Hospital, Boston, Massachusetts, United States., Justus DG; Boston Children's Hospital, Canton, Massachusetts, United States., Armant M; boston children's hospital, boston, Massachusetts, United States., Manis JP; Boston Children's Hospital, Boston, Massachusetts, United States., Williams DA; Boston Children's Hospital, Boston, Massachusetts, United States.
Source: Blood [Blood] 2026 Jun 02. Date of Electronic Publication: 2026 Jun 02.
Publication Type: Journal Article
Journal Info: Publisher: Elsevier Country of Publication: United States NLM ID: 7603509 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1528-0020 (Electronic) Linking ISSN: 00064971 NLM ISO Abbreviation: Blood Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:1528-0020
DOI:10.1182/blood.2026033871