Publications:

Nonsense Suppression Therapy: New Hypothesis for the Treatment of Inherited Bone Marrow Failure Syndromes.

International Journal Of Molecular Sciences

Bezzerri, Valentino V; Api, Martina M; Allegri, Marisole M; Fabrizzi, Benedetta B; Corey, Seth J SJ; Cipolli, Marco M

Publication Date: 2020-06-30

Variant appearance in text: CFTR: G194X

PubMed Link: 32630050

Variant Present in the following documents:

• Main text
• ijms-21-04672.pdf

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Efficacy of Postnatal In Vivo Nonsense Suppression Therapy in a Pax6 Mouse Model of Aniridia.

Molecular Therapy. Nucleic Acids

Wang, Xia X; Gregory-Evans, Kevin K; Wasan, Kishor M KM; Sivak, Olena O; Shan, Xianghong X; Gregory-Evans, Cheryl Y CY

Publication Date: 2017-06-16

Variant appearance in text: CFTR: G194X

PubMed Link: 28624217

Variant Present in the following documents:

• mmc2.pdf
• main.pdf

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Toward postnatal reversal of ocular congenital malformations.

The Journal Of Clinical Investigation

Sahel, José-Alain JA; Marazova, Katia K

Publication Date: 2014-01

Variant appearance in text: CFTR: Gly194X

PubMed Link: 24355915

Variant Present in the following documents:

• Main text

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