Protein / target

Heterogeneous nuclear ribonucleoprotein A1

Encoded byHNRNPA1P09651Homo sapiensSwiss-Prot

UniProt: P09651 Ensembl: ENSG00000135486 HGNC: HGNC:5031 NCBI Gene: 3178

Small-molecule tractable

Druggability

Structure with Ligand

Research papers

Protein at a glance

Biological role

Telomeric repeat-containing RNA binding

Strongest disease association

Amyotrophic Lateral Sclerosis

Via encoding gene HNRNPA1 · Genetic evidence · score 0.78

Research activity

Emerging research

3 papers · latest 2025

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Involved in the packaging of pre-mRNA into hnRNP particles, transport of poly(A) mRNA from the nucleus to the cytoplasm and modulation of splice site selection.

View complete UniProt function annotation

Involved in the packaging of pre-mRNA into hnRNP particles, transport of poly(A) mRNA from the nucleus to the cytoplasm and modulation of splice site selection (PubMed:17371836). Plays a role in the splicing of pyruvate kinase PKM by binding repressively to sequences flanking PKM exon 9, inhibiting exon 9 inclusion and resulting in exon 10 inclusion and production of the PKM M2 isoform (PubMed:20010808). Binds to the IRES and thereby inhibits the translation of the apoptosis protease activating factor APAF1 (PubMed:31498791). May bind to specific miRNA hairpins (PubMed:28431233)

Subcellular location

NucleusCytoplasm

Domains and Gene Ontology detail (34)

Domains & features

RRM 1RRM 2

Gene Ontology

Ccatalytic step 2 spliceosome
Ccytoplasm
Ccytosol
Cextracellular exosome
Cmembrane
Cnucleoplasm
Cnucleus
Cribonucleoprotein complex
Cspliceosomal complex
Csynapse
FDNA binding
FG-rich strand telomeric DNA binding

372 aa · 39 kDa · 3 isoforms

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene HNRNPA1

Gene-level evidence surfaced through the gene HNRNPA1that encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Amyotrophic Lateral Sclerosis

0.83Well supported

Genetic evidence dominant · Open Targets 0.64

Multiple Sclerosis, Chronic Progressive

0.55Moderately supported

Somatic mutation evidence dominant · Open Targets 0.51

Multiple Sclerosis, Relapsing-Remitting

0.37Limited support

Somatic mutation evidence dominant · Open Targets 0.34

Severe Acute Respiratory Syndrome

0.25Preliminary

Pathway evidence dominant · Open Targets 0.37 · no direct causal or clinical evidence

Dengue

0.24Preliminary

Pathway evidence dominant · Open Targets 0.37 · no direct causal or clinical evidence

View evidence synthesis (5)

Amyotrophic Lateral SclerosisWell supported

0.83

agreement 0.71–0.95

Genetic78%Literature11%Animal model11%Genetic literaturedup

Open Targets aggregate 0.64 · 3 independent evidence families · 1 not counted as duplicate

Multiple Sclerosis, Chronic ProgressiveModerately supported

0.55

agreement 0.39–0.71

Somatic mutation98%Literature2%

Open Targets aggregate 0.51 · 2 independent evidence families

Multiple Sclerosis, Relapsing-RemittingLimited support

0.37

agreement 0.18–0.55

Somatic mutation100%

Open Targets aggregate 0.34 · 1 independent evidence family

Severe Acute Respiratory SyndromePreliminary

0.25

agreement 0.07–0.42

Pathway98%Literature2%

Open Targets aggregate 0.37 · 2 independent evidence families · no direct causal or clinical evidence

DenguePreliminary

0.24

agreement 0.01–0.47

Pathway100%

Open Targets aggregate 0.37 · 1 independent evidence family · no direct causal or clinical evidence

The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations

Amyotrophic Lateral Sclerosis0.64

Multiple Sclerosis, Chronic Progressive0.51

Severe Acute Respiratory Syndrome0.37

Dengue0.37

Multiple Sclerosis, Relapsing-Remitting0.34

Tractability

Small molecules — Emerging

Feasibility evidence (structure with ligand and high-quality pocket) — no clinical-stage drug of this modality recorded.

Protein degraders — Emerging

Feasibility evidence (uniprot ubiquitination and database ubiquitination) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (7)

SM · Structure with LigandSM · High-Quality PocketSM · Druggable FamilyPR · UniProt UbiquitinationPR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.