Turning Genes into Medicine: A Closeup of Gene Therapy Drug Discovery

Gene therapies can involve replacing or altering an existing gene. The most common approaches to gene therapy involve AAV viral-vectors that deliver the DNA to cells, liposome encapsulated mRNAs (the same strategy used in some COVID vaccines), or engineering a gene into a patient’s stem cells and re-infusing the cells. This video looks at how the gene editing technology CRISPR and AAVs combine to develop a gene therapy for Duchenne Muscular Dystrophy.

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We are getting to that point but slowly, slower that it should, more urgent work is needed, more phds that encourage research in this area should be open

[tasadaqdugal]

Hi sir we are four brother's and one sister facing lgmd last 5year any treatment for this desace please contact me