Chemical modifications of G418 (geneticin): Synthesis of novel readthrough aminoglycosides results in an improved in vitro safety window but no improvements in vivo.


Journal

Carbohydrate research
ISSN: 1873-426X
Titre abrégé: Carbohydr Res
Pays: Netherlands
ID NLM: 0043535

Informations de publication

Date de publication:
Sep 2020
Historique:
received: 09 03 2020
revised: 17 05 2020
accepted: 01 06 2020
pubmed: 14 7 2020
medline: 1 6 2021
entrez: 14 7 2020
Statut: ppublish

Résumé

G418 is currently the most potent and active aminoglycoside to promote readthrough of eukaryotic nonsense mutations. However, owing to its toxicity G418 cannot be used in vivo to study readthrough activity A robust and scalable method for selective derivatization of G418 was developed to study the biological activity and toxicity of a series of analogs. Despite our synthetic efforts, an improvement in readthrough potency was not achieved. We discovered several analogs that demonstrated reduced zebra fish hair cell toxicity (a surrogate for ototoxicity), but this reduction in cellular toxicity did not translate to reduced in vivo toxicity in rats.

Identifiants

pubmed: 32658832
pii: S0008-6215(20)30150-6
doi: 10.1016/j.carres.2020.108058
pii:
doi:

Substances chimiques

Aminoglycosides 0
Gentamicins 0
antibiotic G 418 A08F5XTI6G

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

108058

Informations de copyright

Copyright © 2020 Elsevier Ltd. All rights reserved.

Auteurs

Ramil Y Baiazitov (RY)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA. Electronic address: rbaiazitov@ptcbio.com.

Westley Friesen (W)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Briana Johnson (B)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Anna Mollin (A)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Josephine Sheedy (J)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Jairo Sierra (J)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Marla Weetall (M)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Arthur Branstrom (A)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Ellen Welch (E)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

Young-Choon Moon (YC)

PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, NJ, 07080, USA.

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Classifications MeSH