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General Biology for Nonmajors Lecture Notes Part 3 - 4.0ed the course!

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Florida State University
Biological Science
BSC 1005

February 21, 2014: Human Germline Engineering • Genetically modified humans? • Gene therapy o This is the process of using DNAas a therapeutic (in contrast to drug therapy) o In general, a mutated gene must be fixed by either  Adding the normal gene  Replacing the mutated gene with a normal gene • Also o Kill cancer cells using genes o DNAvaccines – genes to combat infectious diseases o Somatic cell gene therapy [type 1] vs. reproductive (aka germline) gene therapy [type 2]  Somatic cells: cells derived from the basic germ layers of the body (endoderm, mesoderm, ectoderm) that DO NOT pass their genetic information to the next generation  Germ cells: cells that give rise to the gametes and DO pass their genetic information to the next generation • Gastrulation forms the basic germ layers of the body and creates the germ cells for the next generation. • Two types of delivery for somatic cell gene therapy o Direct delivery o Cell-based delivery  Genetically identical iPSCs are generated from the patient. OR  Adult stem cells are isolated and propagated in the laboratory. This only works for adult stem cells that can be isolated, however.  Gene therapy often exploits viruses to deliver genes to cell nuclei. • Insert a new gene into bits of the virus’DNAand then let the virus perform its normal actions. • Because the vehicles used are viruses, though, there is some risk, because the body might recognize them as invaders and destroy them or develop a strong immune reaction.  Challenges of somatic gene therapy • Must get therapeutic gene into relevant cells’nucleus o Direct vs. cell-based delivery • Genes must be expressed long-term o Foreign genes tend to be “silenced” o Gene repair is not perceived as “foreign” but is much more difficult. • Safety concerns unique to somatic gene therapy o Removal of the engineered cells could prove difficult o 1999 death due to a massive adverse immune response to virus put a huge damper on gene therapy in general o Side effects from integration of foreign DNA o So far, there has been limited success for somatic cell gene therapy o Research in somatic cell gene therapy  Development of gene delivery vehicles that do not rely on viruses  New ways to destroy, introduce, or repair genes • Slide with picture of scissors  Demonstrating treatments in animal models  Using patient-specific iPSCs to correct genetic defects and then creating the appropriate tissue. • Remember – iPSCs can be derived from any adult tissue. They are tricked into thinking they are embryonic and then can be injected into any offending areas.  Reproductive (germline) gene therapy • Gene therapy to alter the genetic constitution of germ cells • Affects all the cells of the body – not targeted to a specific tissue • Manipulates the genome of a person before he/she is born • Imposes a permanent change that is passed on to subsequent generations • Modified version – making “transgenic animals” – animals with an added gene, not a modification to their existing genes o Deliver the gene into the nucleus of a fertilized one cell embryo (zygote) o Develop to a blastocyte and implant it into a uterus o Test to verify that the gene is present in the offspring o To see that it works, florescent proteins are often used – easy to identify. Then, these animals will be florescent if the gene is successfully transferred. o Transgenics work well for “adding” a gene, but to repair or replace a gene currently requires cells that can be cultured in a dish.  To do this, you have to catch the person at the one- cell stage – before he/she really exists.
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