Gene Therapy for Monogenic Diseases

Monogenic diseases, of which there are thousands, are caused by a mutation in a single gene.

CME credit is no longer available for this conference.

Matthew H. Porteus, M.D., Ph.D.

Release Date: 09/10/2006

Upon completion of this Cyberounds^®, you should be able to:

Describe the different types of viral vectors that are being used in gene therapy
Summarize the successes and setbacks in the recent clinical trials for gene therapy
Describe the future directions of gene therapy research to improve the safety of gene therapy.

Genetic Basis of COVID-19 Susceptibility and Severity

Genetically pinpointing who is most at risk from SARS-CoV-2 and who is likely to get the severest infection.

Authors: Cleo Anastassopoulou, Ph.D., and Athanasios Tsakris, M.D., Ph.D., F.R.C.Path.

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Mitochondria play a prominent role in the initiation of apoptosis, an organism's normal and controlled death of cells.

Authors: Claus Desler, Ph.D., and Lene Juel Rasmussen, Ph.D.

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The heritability of SLE is relatively high.

Authors: Chandra Mohan, M.D., Ph.D.

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BRCA1/2 gene mutations are inherited in an autosomal dominant fashion.

Authors: Jennifer Barrick, M.S., C.G.C., Nancie Petrucelli, M.S., C.G.C., and Michael S. Simon, M.D., M.P.H.

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Receptors for sour- and salty-tasting compounds have not been identified, but may be ion channels.

Authors: Hillary L. Shaw, M.D., Cedrick D. Dotson, Ph.D., Steven D. Munger, Ph.D., and Nanette Steinle, M.D.

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