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SUMMARY:Nanotechnology for Targeted in vivo Gene Editing
DTSTART:20140909T141500
DTSTAMP:20260916T043438Z
UID:37f2ab83e8e76fa5601469e5d547053c71860484ccec288ad8a4b6ae
CATEGORIES:Conferences - Seminars
DESCRIPTION:James E. Dahlman\, PhD\, Broad Institute (MIT/Harvard)\, and M
 IT Koch Institute for Cancer Research\, Cambridge\, MA (USA)\nJOINT MATERI
 ALS SCIENCE and BIOENGINEERING SEMINARAbstract:\nOnce viewed as a passive 
 link between DNA and protein\, RNAs are now known to actively and precisel
 y modify gene expression. For example\, siRNAs can reduce the expression o
 f any protein\, while CRISPR-Cas9 enables scientists to make targeted and 
 permanent changes in genomic DNA for the first time.\n \nBecause RNAs can
 \, in theory\, be used to easily manipulate many genes simultaneously\, th
 ey could revolutionize the way we study and treat disease. However\, the f
 ull scientific and clinical potential of RNA is currently limited\, becaus
 e we can not deliver RNA to the right cells in vivo. In vivo RNA delivery
  is difficult\; the molecule must be (a) protected from degradation in the
  bloodstream\, (b) delivered to the correct target tissue\, and (c) ferrie
 d into the right cell\, without setting off an unwanted immune response. W
 hile nanoparticles have delivered RNA to the liver\, delivery to other cel
 l types has remained difficult.\n \nHerein\, I will describe new tools fo
 r in vivo RNA delivery and gene editing\, developed by integrating chemica
 l engineering\, nanotechnology\, biology\, and genomics. One tool\, a nano
 particle named 7C1\, has delivered RNA to the heart and lung at very low d
 oses\, has delivered 5 different RNAs concurrently\, has been used by 10 l
 abs across the United States to study inflammation\, cancer\, heart diseas
 e\, and lung disease\, and is under evaluation for clinical trials in lung
  cancer and heart disease. Surprisingly\, unlike many other nanoparticles\
 , 7C1 nanoparticles do not readily transfect hepatocytes or immune cells i
 n vivo. We believe that this new tool\, which has been described on the co
 ver of Nature Nanotechnology and PNAS\, can be used to easily make targete
 d changes in gene expression.\nI will also describe our recent efforts to 
 generate in vivo DNA deletions using the CRISPR-Cas9 system.Bio:\nJames Da
 hlman is a post-doctoral fellow whose research lies at the interface of en
 gineering\, nanotechnology\, and medicine. He received his PhD from the MI
 T/Harvard Medical School HST program\, where he studied targeted drug deli
 very with Robert Langer. At the end of his PhD\, James had seventeen paper
 s published\, or in various stages of preparation\, including first author
  papers in PNAS\, Nature Reviews Cancer\, and a cover feature in Nature Na
 notechnology. His technology\, which preferentially targets drugs to the l
 ung and heart\, has been used by ten labs across the United States to stud
 y cancer\, atherosclerosis\, inflammation\, emphysema\, and pulmonary hype
 rtension\, and is being evaluated for clinical trials in lung cancer and c
 ardiovascular disease. More recently\, James invented a platform that will
  enable scientists to study thousands of clinical drug delivery vehicles i
 n vivo (currently\, only a few candidates are normally tested in vivo). He
  is currently studying in vivo genome editing using CRISPR-Cas9 with Feng 
 Zhang at the Broad Institute\; his research will combine genomics\, drug d
 elivery\, nanotechnology\, and RNA\, with the goal of radically improving 
 the way we study gene function.\nJames has given nearly thirty presentatio
 ns\, including invited talks at Harvard and in Europe\, has received numer
 ous awards for his work as a PhD student\, and was likely the only student
  in the United States to simultaneously win the NSF\, NDSEG\, MIT Presiden
 tial\, Whitaker\, and NIH Oxford Cambridge Fellowships in 2009.
LOCATION:SV1717a http://map.epfl.ch/?room=sv1717a
STATUS:CONFIRMED
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