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infants<00:00:01.439> born<00:00:01.760> with<00:00:01.920> the<00:00:02.040> most<00:00:02.320> severe<00:00:02.760> form

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infants born with the most severe form
 
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infants born with the most severe form
of<00:00:03.240> SMA<00:00:03.959> often<00:00:04.240> don't<00:00:04.480> make<00:00:04.640> it<00:00:04.759> to<00:00:04.920> their

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of SMA often don't make it to their
 
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of SMA often don't make it to their
second<00:00:05.919> birthday<00:00:06.919> but<00:00:07.120> researchers<00:00:07.720> have

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second birthday but researchers have
 
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second birthday but researchers have
been<00:00:08.080> studying<00:00:08.480> how<00:00:08.599> to<00:00:08.800> fix<00:00:09.080> this<00:00:09.240> splicing

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been studying how to fix this splicing
 
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been studying how to fix this spliacing
problem<00:00:12.840> one<00:00:13.120> solution<00:00:13.679> is<00:00:13.920> called<00:00:14.599> Nusinersen<00:00:15.599> it

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problem one solution is called Nusinersen it
 
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problem one solution is called Nusinersen it
enables<00:00:16.359> SMA<00:00:16.880> patients<00:00:17.320> to<00:00:17.520> make<00:00:17.880> more<00:00:18.279> SMN

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enables SMA patients to make more SMN
 
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enables SMA patients to make more SMN
protein<00:00:21.000> but

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protein but
 
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protein but
how<00:00:23.279> Nusinersen<00:00:24.359> is<00:00:24.599> a<00:00:24.800> short<00:00:25.199> sequence<00:00:25.640> of<00:00:25.840> RNA

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how Nusinersen is a short sequence of RNA
 
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how Nusinersen is a short sequence of RNA
that<00:00:26.560> binds<00:00:27.160> just<00:00:27.519> after<00:00:27.880> Exxon<00:00:28.320> 7<00:00:29.039> in<00:00:29.199> the

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that binds just after Exxon 7 in the
 
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that binds just after Exxon 7 in the
unedit<00:00:30.279> RNA

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unedit RNA
 
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unedit RNA
message<00:00:32.439> this<00:00:32.680> tiny<00:00:33.000> piece<00:00:33.200> of<00:00:33.360> RNA<00:00:33.920> prevents

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message this tiny piece of RNA prevents
 
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message this tiny piece of RNA prevents
Exxon<00:00:34.879> 7<00:00:35.239> from<00:00:35.440> being<00:00:36.200> skipped<00:00:37.200> it<00:00:37.320> is<00:00:37.600> called

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Exxon 7 from being skipped it is called
 
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Exxon 7 from being skipped it is called
an<00:00:38.200> anti-sense<00:00:39.280> oligonucleotide<00:00:40.280> or

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an anti-sense oligonucleotide or
 
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an anti-sense oligonucleotide or
ASO<00:00:42.680> Exon<00:00:43.120> 7<00:00:43.600> is<00:00:43.760> now<00:00:44.000> included<00:00:44.920> in<00:00:45.200> the<00:00:45.399> edited

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ASO Exon 7 is now included in the edited
 
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ASO Exon 7 is now included in the edited
RNA<00:00:46.320> message<00:00:46.960> which<00:00:47.199> in<00:00:47.360> turn<00:00:47.800> generates

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RNA message which in turn generates
 
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RNA message which in turn generates
fulllength<00:00:49.600> functional<00:00:50.199> SMN<00:00:50.520> 

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protein<00:00:54.640> by<00:00:54.840> preventing<00:00:55.280> xon<00:00:55.719> 7<00:00:56.039> from<00:00:56.239> being

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protein by preventing xon 7 from being
 
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protein by preventing xon 7 from being
skipped<00:00:57.000> Nusinersen<00:00:57.760> reverses<00:00:58.320> symptoms<00:00:58.840> in<00:00:59.039> SMA

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skipped Nusinersen reverses symptoms in SMA

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skipped Nusinersen reverses symptoms in SMA
patients