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LAMP5 shRNA

Development stage
Preclinical
Lead developer
University of Texas MD Anderson Cancer Center
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Intratumoral, Intraperitoneal
01

Overview

LAMP5 shRNA is a research-grade RNA interference (RNAi) tool designed to knock down the expression of the Lysosomal Associated Membrane Protein Family Member 5 (LAMP5) gene. It functions by delivering a short hairpin RNA construct that targets the LAMP5 mRNA transcript, leading to its degradation and a subsequent reduction in LAMP5 protein levels. In preclinical models of high-risk multiple myeloma and MLL-rearranged acute leukemia, LAMP5 knockdown has been shown to inhibit tumor cell growth, induce apoptosis, and dysregulate critical pathways such as the G2M checkpoint, mTORC1 signaling, and autophagy. While it serves as a valuable tool for validating LAMP5 as a potential therapeutic target, it is not a clinical-stage drug and is primarily used by academic research groups for mechanistic studies.

Other names
LAMP5 short hairpin RNALAMP-5 short hairpin RNALAMP 5 short hairpin RNA
02

Targets

LAMP5 (Lysosomal-associated membrane protein 5)

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