Target intelligence / Profile preview

Long intergenic non-protein coding RNA 1257 (LINC01257)

Target
LINC01257
Molecular classification
Long non-coding RNA (lncRNA), Non-protein coding RNA, Other (does not fall under classic ‘receptor’, ‘enzyme’, etc.)
01

Overview

Long intergenic non-protein coding RNA 1257 (LINC01257) is a long non-coding RNA (lncRNA) gene that does not code for any protein but is transcribed as an RNA molecule longer than 200 nucleotides. It is specifically and highly expressed in the t(8;21) subtype of pediatric acute myeloid leukemia, but absent in healthy hematopoietic cells and other AML subtypes. LINC01257's expression promotes leukemia cell proliferation and survival, suggesting an oncogenic role. Therapeutic silencing using siRNA-loaded lipid nanoparticles (LNP-si-LINC01257) reduces AML cell proliferation and viability in vitro, while showing minimal toxicity in healthy cells, supporting its relevance as a highly disease-specific and promising RNA-based therapeutic target. As a lncRNA, LINC01257 is involved in gene regulation, chromatin modification, and possibly signaling pathways that underpin leukemogenesis. There are currently no known clinical drugs targeting LINC01257 beyond experimental siRNA-based approaches. Elevated levels of LINC01257 can serve as a biomarker for diagnosis, prognosis, or stratification of AML patients with the t(8;21) translocation.

Other names
LINC01257Long intergenic non-protein coding RNA 1257ENSG00000204603
02

Mechanism of action

RNA interference (RNAi): siRNA delivery leads to sequence-specific silencing of LINC01257 transcript. Silencing LINC01257 impairs AML cell proliferation and survival.

03

Biological functions

Regulation of gene expressionChromatin modificationCell cycle regulationPromotion of cell proliferation and leukemia survival (specifically in t(8;21) AML)
04

Disease associations

Cancer (specifically, acute myeloid leukemia subtype t(8;21))
05

Safety considerations

Delivery challenges: as a lncRNA, targeting requires advanced RNA technologies with specific delivery mechanisms (e.g., lipid nanoparticles).Off-target effects are limited due to disease-selective expression, but general concerns for RNAi therapeutics remain (e.g., immune response, delivery efficiency).
06

Interacting drugs

siRNA-based therapeutics (experimental, not small molecules)

1 more in the full profile.

07

Biomarkers

High expression of LINC01257 in t(8;21) AML (acute myeloid leukemia) cells is associated with poor prognosis and increased proliferationDisease-specific upregulation in t(8;21) AML relative to other AML subtypes and healthy cells

Beyond the preview

Go deeper on Long intergenic non-protein coding RNA 1257 (LINC01257).

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Long intergenic non-protein coding RNA 1257 (LINC01257).

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call