Understanding Gene Editing

Transcript

• 00:05Gene is a piece of
• 00:06the DNA we call blueprint
• 00:08for life.
• 00:10DNA or gene give instruction
• 00:13that goes to the mRNA,
• 00:14then mRNA
• 00:15translate to the protein.
• 00:18The protein pretty much responsible
• 00:20for all our biological
• 00:22functions.
• 00:23That's in how we work
• 00:24because our protein.
• 00:26Our gene determines which protein
• 00:28to be expressed.
• 00:30Every cell you have to
• 00:31have
• 00:32a sort of DNA
• 00:34to make the cell functions.
• 00:36But cells in different organs,
• 00:37different tissues, they
• 00:39express certain subset of genes.
• 00:44The genetic variant can cause
• 00:46called a personal trait,
• 00:48which is neutral benign.
• 00:50But some of them are
• 00:51crucial for our health. If
• 00:53that particular variant
• 00:55end up in a very
• 00:56critical part of the gene
• 00:58We may produce a faulty
• 01:00protein
• 01:01or, you know, we cannot
• 01:03produce a specific protein at
• 01:05all.
• 01:06That became disease causing. Oftentimes,
• 01:09we commutation.
• 01:10For some genetic disease, you
• 01:12know,
• 01:12the cause could be very
• 01:14complicated. Without understanding the mechanism,
• 01:16it's certainly very difficult to
• 01:18design any, treatment.
• 01:23Gene editing can modulate or
• 01:25correct a gene,
• 01:27which you believe in causing
• 01:28disease.
• 01:29It's a very powerful tool
• 01:31which could be used to
• 01:32produce the
• 01:33the right form of protein,
• 01:35which respond for most, biological
• 01:38functions.
• 01:39Before, there is no tool
• 01:42can do this job very,
• 01:43very efficiently.
• 01:45Even somebody carries a genetic
• 01:47variance,
• 01:48this genetic variance may not
• 01:49affect all cells or tissues.
• 01:52When CRISPR coming, so this
• 01:55became a reality.
• 01:57So you can editing or
• 01:58correct the gene very efficient
• 02:00way.
• 02:01Basically, you can target at
• 02:03the specific cells.
• 02:04So there's a lot of
• 02:05advantages that makes CRISPR,
• 02:08so revolutionary.
• 02:09And then, you know, now
• 02:10a lot of, people, including
• 02:12us,
• 02:13try to use this technology
• 02:15to, you know, to treat
• 02:16genetic disease.
• 02:22Pretty much the biological structure
• 02:24of the brain makes drug
• 02:25delivery to the brain. This
• 02:26case deliver genome medicines to
• 02:28the brain. Very, very challenging.
• 02:31This very innovative technology,
• 02:33in short we call STAP,
• 02:34allow
• 02:35us to deliver
• 02:37the crisp editing
• 02:38more efficient
• 02:40to the brain.
• 02:42Basically, it's a technology based
• 02:43on the use of small
• 02:45molecules to deliver protein based
• 02:47genome editing machinery.
• 02:50We have developed a thousand
• 02:52of stuff molecules already.
• 02:54So we screen those stuff
• 02:56molecules.
• 02:57We found certain stuff molecules
• 02:59like a specific cell type.
• 03:02They enable,
• 03:03deliver all the CRISPR machinery
• 03:06into the brain and dispose
• 03:08the swabs brain. So that's
• 03:09very unique.
• 03:12We have five thousand disease.
• 03:13We know the mechanism,
• 03:14but
• 03:15each one may have a
• 03:16different mechanism. So therefore,
• 03:19individual disease, you may have
• 03:22developed individual
• 03:23sort of for the tool,
• 03:24even under the umbrella for
• 03:26the CRISPR editing.
• 03:28So far, I think we
• 03:29used the Cas9
• 03:31for the disease we're working
• 03:32on right now. It's Angelman
• 03:34syndrome. That potentially can
• 03:36apply to a few hundred
• 03:38other new genetic disease.
• 03:41Our hope is that in
• 03:42the next few years with
• 03:44the support from NIOSH,
• 03:46we can move this stuff
• 03:47based therapy into clinic.
• 03:51But stuff technology is a
• 03:52platform technology,
• 03:54which means that we can,
• 03:56potentially adapt this technology,
• 03:58for deliver our other, payloads
• 04:01to other cells and tissues.
• 04:05That means in long term,
• 04:06we hope, this technology
• 04:08can be repurposed enough for
• 04:10treatment of many other rare
• 04:11disease.