> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/target/smn1-cca81f7f-af07-5c1a-9fe5-f974cdba6839
> Record changed: 2026-10-13

# SMN1

ENSG00000172062

Target class

Epigenetic regulator

Chromosome

5

Approved drugs

1

Clinical candidates (approved and in development)

1

## Clinical evidence

**Approved drugs**

1

**Clinical candidates (approved and in development)**

1

## Druggability

**Chemical probe available**

Yes

## Names

**Target name**

SMN1

**Gene name**

Survival of motor neuron 1, telomeric

**Gene symbol**

SMN1

**Protein name**

Survival motor neuron protein (Component of gems 1) (Gemin-1)

### Other names (7)

- BCD541
- SMNT
- SMA1
- SMA2
- SMA3
- GEMIN1
- TDRD16A

### Previous gene symbols (2)

- SMA

## Classification

**Gene biotype**

Protein coding

**Gene family**

Tudor domain containing|SMN complex|Proteins encoded by multiple genes

**Target class**

Epigenetic regulator

**Target type**

Human gene

## Expression and localisation

**Tissues expressed in**

53

**Protein localisation evidence grade**

Supported

**Highest tissue expression**

8.25

**Tissue specificity (Gini)**

0.244

**Tissue specificity (tau)**

0.517

## Pathways and interactions

**Protein interaction confidence**

0.87316

**Protein interaction partners**

50

### KEGG gene identifiers (2)

- hsa:6606
- hsa:6607

### Reactome pathways (2)

- R-HSA-191859
- R-HSA-9754678

## Genomic location

**End position**

70953942

**Start position**

70925030

**Chromosome**

5

**Strand**

Forward

## Identifiers

**Ensembl gene ID**

ENSG00000172062

**HGNC ID**

HGNC:11117

Source: [www.genenames.org](https://www.genenames.org/data/gene-symbol-report/#!/hgnc%5Fid/HGNC:11117)

**NCBI Gene ID**

6606

**UniProt ID**

Q16637

## Cellular location

- Nucleus, gem . Nucleus, Cajal body . Cytoplasm . Cytoplasmic granule . Perikaryon . Cell projection, neuron projection . Cell projection, axon . Cytoplasm, myofibril, sarcomere, Z line . Note=Colocalizes with actin and at the Z-line of skeletal muscle (By similarity). Under stress conditions colocalizes with RPP20/POP7 in punctuated cytoplasmic granules (PubMed:14715275). Colocalized and redistributed with ZPR1 from the cytoplasm to nuclear gems (Gemini of coiled bodies) and Cajal bodies (PubMed:11283611). Colocalizes with FMR1 in cytoplasmic granules in the soma and neurite cell processes (PubMed:18093976). .
- Cytosol
- Nuclear bodies

### Publication evidence (6)

- PubMed 11283611  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/11283611/)
- PubMed 8670859  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/8670859/)
- PubMed 12067652  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/12067652/)
- PubMed 21072240  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/21072240/)
- PubMed 14715275  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/14715275/)
- PubMed 18093976  
Source: [pubmed.ncbi.nlm.nih.gov](https://pubmed.ncbi.nlm.nih.gov/18093976/)

## Tissue expression

**Tissue specificity (tau)**

0.517439

**Expression inequality (Gini)**

0.244496

### Highest expression

Expression in transcripts per million

**Ovary**

8.25

**Cells - EBV-transformed lymphocytes**

7.77

**Cervix - Ectocervix**

7.7

**Cervix - Endocervix**

7.57

**Uterus**

6.82

### All tissues (54)

Expression in transcripts per million

- Ovary8.25
- Cells - EBV-transformed lymphocytes7.77
- Cervix - Ectocervix7.7
- Cervix - Endocervix7.57
- Uterus6.82
- Fallopian Tube6.52
- Nerve - Tibial6.44
- Testis5.98
- Thyroid5.61
- Vagina5.59
- Cells - Cultured fibroblasts5.55
- Brain - Cerebellar Hemisphere5.49
- Pituitary5.46
- Prostate5.21
- Artery - Tibial4.99
- Bladder4.98
- Breast - Mammary Tissue4.86
- Adipose - Visceral (Omentum)4.75
- Colon - Sigmoid4.61
- Adipose - Subcutaneous4.58
- Lung4.55
- Artery - Coronary4.39
- Artery - Aorta4.3
- Skin - Not Sun Exposed (Suprapubic)4.24
- Brain - Cerebellum4.12

And 29 more, listed on the interactive page.

## Biological functions

### Biological processes (3)

- GO:0000245  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0000245)
- GO:0000387  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0000387)
- GO:0006353  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0006353)

### Cellular components (12)

- GO:0005634  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0005634)
- GO:0005654  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0005654)
- GO:0005737  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0005737)
- GO:0005829  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0005829)
- GO:0015030  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0015030)
- GO:0016604  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0016604)
- GO:0032797  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0032797)
- GO:0034719  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0034719)
- GO:0036464  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0036464)
- GO:0043005  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0043005)
- GO:0043204  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0043204)
- GO:0097504  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0097504)

### Molecular functions (2)

- GO:0005515  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0005515)
- GO:0042802  
Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/QuickGO/term/GO:0042802)

## Related records

### Trials (111)

- A Clinical Study of SHR-9839 as Monotherapy or in Combination With Chemotherapy Plus Adebrelimab Versus Chemotherapy Plus Adebrelimab in Patients With Advanced Esophageal Squamous Cell Carcinoma
- A Clinical Trial to Evaluate Efficacy and Safety of Two Different Formulations of Topical Calcipotriol in Patients With Mild to Moderate Plaque Psoriasis
- A Clinical Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of PGDM1400LS Alone and in Combination With VRC07-523LS and PGT121.414.LS in Healthy, HIV-uninfected Adult Participants
- A IB/II Phase Clinical Study on the Safety, Tolerability, and Efficacy of SHR-9839(sc) in Combination Anti-tumor Therapy in Patients With Solid Tumors
- A Long Term Roll-over Study to Investigate the Efficacy and Safety of Adalimumab Given Long-term to Patients With Rheumatoid Arthritis
- A Master Protocol (IIBD): A Study of Multiple Drugs in Adults With Ulcerative Colitis or Crohn's Disease
- A Phase I Clinical Study of ART101 Injection in Healthy Adult Subjects
- A Phase IB/II Clinical Study of SHR-9839 for Injection Combined With Other Anti-tumor Therapies in Patients With Advanced Solid Tumors
- [A Phase II Clinical Study of HRS-7058 in Combination With Antitumor Drugs in Patients With Advanced Malignant Tumour](https://trialight.com/data/trial/a-phase-ii-clinical-study-of-hrs-7058-in-combination-with-66a0a475-49b9-4ee9-a803-aeb14e30897a.md)
- A Phase II Randomized Study Comparing the Efficacy and Safety of Targeted Therapy or Cancer Immunotherapy Versus Platinum-Based Chemotherapy in Patients With Cancer of Unknown Primary Site
- A Phase III Study in Subjects With Mild to Moderate Psoriasis.
- A Pilot Study of Boceprevir for the Treatment of Genotype 6 HCV
- A Prospective Randomized Double-Blinded Placebo Controlled, Explorative Phase I Trial to Investigate the Safety and Tolerability of Two Different Doses of Topically Administered APOSEC™ in Healthy Male Subjects With Artificial Dermal Wounds
- A Psoriasis Plaque Test Study With LEO 90100 Cutaneous Spray, Ointment, in Psoriasis Vulgaris
- A Single Dose Pharmacokinetics (PK) Study of GSK2434735 in Healthy Male Volunteers
- A Study of BGM0504 Injection in Participants with Obesity or Overweight
- A Study of Daratumumab With the Addition of Recombinant Human Hyaluronidase (rHuPH20) for the Treatment of Participants With Relapsed or Refractory Multiple Myeloma
- A Study of Efficacy and Safety of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Ulcerative Colitis
- A Study of Guselkumab in Participants With Active Lupus Nephritis
- A Study of Guselkumab in Participants With Systemic Sclerosis

### Drugs (1)

- [Onasemnogene abeparvovec](https://trialight.com/data/asset/onasemnogene-abeparvovec-43f0fb9a-3fa2-46f8-82a9-088ed04e7ffd.md) Survival motor neuron protein exogenous gene

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