11BIS1012013MicroRNALect11

Adm is seen in adults 15 million people in the united

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Unformatted text preview: ns and RNAs Biological RNAs MirRNA Therapeutics COMPANY OVERVIEW: FIRST IN HUMAN CLINICAL TRIALS for a chemically optimized siRNA — a major milestone in the development of RNAibased therapeutics. Sirna-027, for the treatment of the wet-form of age-related macular degeneration (AMD), a condition in which the cells of the macula lutea degenerate, resulting in blurred vision and ultimately blindness. The macula is a small area at the back of the eye, near the center of the retina, at which visual perception is most acute. AMD occurs most often with increasing age (age-related macular degeneration or AMD), but there are hereditary forms of the disease that affect children and teenagers as well. ADM is seen in adults 15 million people in the United States over 60 years of age. Our therapeutic programs encompass a broad range of therapeutic areas including: Anti-Viral, Dermatology, Metabolism, Neurology, Ocular, Oncology, and Respiratory. Specific preclinical and clinical programs include: viral hepatitis, an infection of the liver that is caused by a variety of RNA viruses (hepatitis A virus, hepatitis B virus, hepatitis C virus, etc.).(HBV & HCV), hair removal, asthma and COPD, diabetes, Huntington’s disease, and oncology. These programs represent large market opportunities and significant unmet medical needs. BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013 BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 17 Flavr Savr Tomatoe and Antisense* Flavr ­ Antisense RNA methods have been used for Antisense commercial food production. The Flavr Savr Flavr tomato was developed by Calgene Inc. of Davis, Calgene California in 1991 and was approved by the U.S. FDA in 1994. The tomato was the first whole food created by biotechnology that was evaluated by the FDA. One of the problems associated with tomato farming is that the fruit must be picked while still green in order to be shipped to market without being crushed. The enzyme that causes softening in tomatoes is polygalacturonase (PG). polygalacturonase This enzyme breaks down pectin as the tomato ripens, leading to a softer fruit. Calgene suppressed the expression of the gene encoding PG by introducing a gene encoding the antisense strand of the mRNA. When the introduced gene was expressed, the antisense strand bound to the PG mRNA, suppressing the translation of the enzyme. The Flavr Savr tomatoes therefore had low PG levels and remained firmer when ripe. This meant the Flavr Savr tomatoes could be ripen on the vine and then be shipped to market. Although the Flavr Savr tomatoes were approved for sale in the U.S., production problems and consumer wariness stopped the production of this fruit in 1997. fruit BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013 BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 18 Summary ­ ­ ­ ­ Transcription can be “cosuppressed” or “knocked down” by Transcription small interfering RNAs (siRNAs), complementary to the mRNA small Although it was thought that a single stranded anti-sense RNA Although was responsible for cosuppression, we now know that a dsRNA is needed. is Extensive processing of the dsRNA is needed to produce the Extensive siRNA. siRNA can inhibit gene expression by promoting ribonuclease degradation or blocking the translation of the mRNA target. mRNA This whole process is called RNA interference (RNAi) and it has great potential for health and agriculture. has BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013 BIS101­001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 19...
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