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Targeting FSHD With Designed Antisense RNAs
Targeting FSHD With Designed Antisense RNAs 1024 683 Abbie Roth

Researchers describe small nuclear RNA antisense expression cassettes as a way to silence the DUX4 gene underlying facioscapulohumeral muscular dystrophy. Facioscapulohumeral muscular dystrophy (FSHD) arises from genetic and epigenetic changes that result in expression of the DUX4 gene in muscle. DUX4 is a developmental gene that is normally off in healthy muscle.  When expressed in…

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How to Make a Viral Vector
How to Make a Viral Vector 150 150 Abbie Roth

The Research Institute at Nationwide Children’s Hospital is the home to a current Good Manufacturing Practices (cGMP) Clinical Manufacturing Facility (CMF) that operates according to FDA cGMP Guidelines to ensure the safety of manufactured biologic products. The Space The CMF is a 9000-sq-ft space, including a 7500-sq-ft clean room suite with ISO Class 5/7/8 spaces…

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