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ABCB5-shRNA

Development stage
Preclinical
Lead developer
Boston Children's Hospital
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

ABCB5-shRNA is a short hairpin RNA (shRNA) therapeutic candidate and research tool designed to silence the expression of the ATP-binding cassette transporter ABCB5. ABCB5 has been identified as a critical cell-surface marker for malignant melanoma initiating cells (MMICs) and plays a functional role in melanoma tumorigenic growth and progression. By utilizing RNA interference (RNAi), ABCB5-shRNA knocks down the expression of this transporter, thereby impairing the ability of melanoma cells to maintain efficient tumor growth. Research presented at the AACR 2013 meeting by investigators from Boston Children's Hospital and Brigham and Women's Hospital demonstrated that ABCB5-shRNA knockdown significantly downregulates tumor progression in immunocompromised mouse models, suggesting that ABCB5 is a master regulator of melanoma and a viable therapeutic target.

Other names
ABCB5 short hairpin RNAABCB-5 short hairpin RNAABCB 5 short hairpin RNA
02

Targets

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