Gene Therapy by Rachel Contich https://padlet.com/rcontich/hb35af9raz68 Made with ♥ en-us 2018-02-05 17:27:11 UTC 2018-02-05 17:53:45 UTC hello@padlet.com tknierim1 https://padlet.com/rcontich/hb35af9raz68/wish/228244715 Gene Therapy is a way to fix a defective gene by adding in a correct form of that gene. It uses vectors to transfer the gene into the body, but not all genetic problems can be solved through gene therapy. ]]> 2018-02-05 17:34:07 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228244715 Tools to Trade ghicks6 https://padlet.com/rcontich/hb35af9raz68/wish/228249700 Gene delivery:  requires an effective way to get DNA into cells.Hardest part is finding a suitable vector for treating a disorder.A vector has to target the right cell, integrate the gene in the cell, and activate  the gene. 
Viral Vectors: advantages of the viral vectors are they are good at targeting and entering cells, can determine specific cells and they can be modified so that they do not replicate and destroy cells. The disadvantages are they can only carry limited amount of genetic material and they can cause immune responses in patients that may cause them to gt sick.

]]> 2018-02-05 17:43:01 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228249700 Repairing Mutations mbouffard1 https://padlet.com/rcontich/hb35af9raz68/wish/228250295 Replacing a defective copy of a gene with a working copy. 
(SMaRT)- Spliceosome-Mediated RNA Trans-splicing
This technique targets and repairs the mRNA transcripts copied from the mutated gene and repairs the damaged section of DNA.
This gene editing technique uses enzymes designed to target specific DNA sequences. The enzymes cut out the faulty sequence and replace it with a functional copy.

]]> 2018-02-05 17:44:05 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228250295 Dominant Negative rcontich https://padlet.com/rcontich/hb35af9raz68/wish/228250603 Some gene mutations lead to the production of a dominant negative protein. This protein potentially blocks the protein from functioning. And in this case, adding an additional copy will not help because the dominant negative protein will still not function correctly.]]> 2018-02-05 17:44:40 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228250603 Gene Silencing mbouffard1 https://padlet.com/rcontich/hb35af9raz68/wish/228252303 Gene silencing is an approach used to turn a gene "off" so that no protein is made from it.
Triple-helix-forming oligonucleotide gene therapy targets the DNA sequence of a mutated gene to prevent its transcription.
RNA Interference- The short piece of RNA will find and attach to its complementary sequence, forming a double-stranded RNA molecule, which the cell then destroys.
Ribozyme gene therapy- ribozymes are designed to find and destroy mRNA encoded by the mutated gene so that no protein can be made from it]]> 2018-02-05 17:47:34 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228252303 Gain-of-function rcontich https://padlet.com/rcontich/hb35af9raz68/wish/228253233 A gain of function mutation is when the altered gene product has a new molecular function/gene expression]]> 2018-02-05 17:49:06 UTC https://padlet.com/rcontich/hb35af9raz68/wish/228253233