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

Nonsense mutations leading to premature stop codons are common occurring in approximately 12% of all human genetic diseases. Thus, pharmacological nonsense mutation suppression strategies would be beneficial to a large number of patients if the drugs could be targeted to the affected tissues at the...

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Main Authors: Xia Wang (Author), Kevin Gregory-Evans (Author), Kishor M. Wasan (Author), Olena Sivak (Author), Xianghong Shan (Author), Cheryl Y. Gregory-Evans (Author)
Format: Book
Published: Elsevier, 2017-06-01T00:00:00Z.
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