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AAV6 Vector Production and Purification for Muscle Gene Therapy.

Jeffrey S. Chamberlain 1. Christine L. Full text PDF Related articles. Abstract Vectors derived from adeno-associated viruses AAV have been generated using numerous naturally occurring and synthetic serotypes of the virus. Citations 3 Recent citations: Julian N. Ramos et al. Related articles Based on techniques. Wu et al.

AAV6 Vector Production and Purification for Muscle Gene Therapy.

References Emery AE The muscular dystrophies. Mol Ther — Grimm D, Kay MA, Kleinschmidt JA Helper virus-free, optically controllable, and two-plasmid-based production of adeno-associated virus vectors of serotypes 1 to 6. Additionally, the Gene Therapy Initiative intentionally contains an educational component as, while promising, gene therapy is new and presents different questions and considerations than those of previous therapies. Gene therapy for Duchenne is centered on the goal of successfully introducing a smaller, but efficient version of dystrophin into the muscle cell. The most logical protein to introduce is dystrophin, the protein that is missing in Duchenne. Because dystrophin is such a large protein, smaller versions, referred to as micro-dystrophins are inserted into the delivery vehicle which is typically a virus.

Other muscle stabilizing proteins, such as follistatin, GALGT2, biglycan, and others can also be introduced through gene therapy with the aim of compensating for the missing dystrophin. Scientists had to find a way to deliver these proteins into muscle cells.

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This was challenging because our bodies are made up of so many muscles, many of which are difficult to reach. Over many years of painstaking research, scientists have found a virus that infects muscle cells.

They removed the qualities or genes that make humans sick a term known as gutting the virus. Researchers today are using an adeno-associated virus , often referred to as AAV , as viruses have evolved over time to deposit their own genetic code into cells. While this may seem simple, this is a big moment for gene therapy, even outside of the Duchenne community. The DNA then heals, and a modified dystrophin production is restored.

This is often thought of as permanent exon skipping. Jerry Mendell and Dr. To date, only a few patients have been dosed. This grant brought focus to essential questions that have to be examined. In other words, to ensure safety, we have to make sure the molecular scissors targets only the dystrophin gene.

Through this grant, Dr. Olson examined the stability and functionality of edited forms of dystrophin, assessing possible off-target effects in mouse models, developed for this specific question, in order to determine the safety of this approach.


PPMD awarded Dr. This is an important next step in our exploration of both gene therapy and exon skipping as potential treatments for Duchenne. To better understand the role of the immune system on dystrophin restoration, Dr. Nagaraju will explore the combination of exon skipping or micro-dystrophin gene therapy with an immunosuppressive treatment, using one of the following drugs:. Presumably this region is important for protecting cardiac function.