Gentamicin

Aminoglycoside antibiotic capable of translational readthrough

Phase of research

FDA-/EMA-approved for Other Purpose

How it helps

Restore CFTR Function

General information

Gentamicin is a broad-spectrum aminoglycoside antibiotic made from Micromonospora purpurea. It was approved for medical use in 1964 and is used to treat several types of bacterial infections, including bone infections, endocarditis, pelvic inflammatory disease, meningitis, pneumonia, urinary tract infections, and sepsis. In relation to cystic fibrosis, our AIM tool found the data that gentamicin can help nonsense CFTR mutations, such as W1282X, G542X, R553X, and Y122X, by suppressing their premature termination signals, which leads to the expression of full-length CFTR protein at the cell membrane.

Gentamicin on PubChem
Gentamicin on DrugBank
Gentamicin on Wikipedia

Synonyms

gentamicin sulfate

Marketed as

N/A

Dietary sources

N/A

Structure image not available

C₂₁H₄₃N₅O₇

Drug-Mutation Relation

results for R352Q / G542X

See all data on Gentamicin

Treats

Mutation	Link	Tested on	Impact factor	Notes
G542X	Suppression of CFTR premature termination codons and rescue of CFTR protein and function by the synthetic aminoglycoside NB54.	human cell cultures	4.67	NB54 has similar effect but is less toxic.
G542X	Aminoglycoside suppression of a premature stop mutation in a Cftr-/- mouse carrying a human CFTR-G542X transgene.	mice	3.30
G542X	Suppression of a CFTR premature stop mutation in a bronchial epithelial cell line.	human cell cultures	28.14
G542X	Clinical doses of amikacin provide more effective suppression of the human CFTR-G542X stop mutation than gentamicin in a transgenic CF mouse model.	mice	5.16	Less effective and more toxic than amikacin.

Does not treat

Mutation	Link	Tested on	Impact factor	Notes