Medical Professional Publications

Research Now: Rodino-Klapac’s Lab is Investigating Viral Vector Delivery of Large Genes in Pieces, Which Later Recombine

Dr. Rodino-Klapac’s research is focused on developing treatments for Duchenne Muscular Dystrophy (DMD) and Limb Girdle Muscular Dystrophies (LGMDs) using recombinant adeno-associated virus (rAAV). Her laboratory is investigating novel approaches to deliver large genes using viral vectors that encapsulate the gene in pieces that later recombine to generate a full length protein. They have shown proof of principle for this approach in an animal model for LGMD2B (dysferlin deficiency) and are now in the process of translating this therapy to the bedside. Utilizing a different approach for treatment of DMD, they demonstrated that co-delivery of muscle modulating proteins such as Follistatin and Alpha7-Integrin along with a miniature dystrophin gene (Micro-dystrophin) results in greater functional outcomes over single gene replacement in pre-clinical studies. These studies have been supported by the Jain Foundation and Jesse’s Journey Foundation.

Related Publications:

Rodino-Klapac LR, Janssen PML, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark, KR, Sahenk Z, Mendell JR, Kaspar BK. Micro-dystrophin and Follistatin Co-Delivery Restores Muscle Function in Aged DMD Model. Human Molecular Genetics. 2013 Jul 30. [Epub ahead of print]

Heller KN, Montgomery CL, Janssen PML, Clark KR, Mendell JR, Rodino-Klapac LR. AAV mediated overexpression of Human α7 Integrin leads to histological and functional improvement in dystrophic mice. Molecular Therapy. 2013 Mar, 21(3):520-5.

Rodino-Klapac LR, Mendell JR, Sahenk Z. Update on the Treatment of Duchenne Muscular Dystrophy. Current Neurology and Neuroscience Reports. 2013 Mar, 13(3):332.

Grose WG, Clark KR, Griffin DG, Shontz K, Malik V, Montgomery CL, Janssen, PM, Brown RJ, Mendell JR, Rodino-Klapac LR. Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency Following AAV5 Gene Transfer. PLoS One. 2012 Jun 15, 7(6):e39233.

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