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let's<00:00:01.480> say<00:00:01.680> The<00:00:01.839> SMN<00:00:02.360> Gene<00:00:02.800> generates<00:00:03.320> RNA

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let's say The SMN Gene generates RNA
 
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let's say The SMN Gene generates RNA
copies<00:00:04.240> made<00:00:04.440> up<00:00:04.759> of<00:00:04.960> introns<00:00:05.640> and<00:00:05.839> exons<00:00:06.440> like

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copies made up of introns and exons like
 
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copies made up of introns and exons like
this<00:00:08.200> normal<00:00:08.519> RNA<00:00:08.960> splicing<00:00:09.400> would<00:00:09.559> delete

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this normal RNA splicing would delete
 
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this normal RNA splicing would delete
the<00:00:10.000> introns<00:00:10.759> the<00:00:10.880> gibberish<00:00:11.440> letters<00:00:11.840> in

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the introns the gibberish letters in
 
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the introns the gibberish letters in
blue<00:00:12.840> and<00:00:12.960> leave<00:00:13.320> only<00:00:13.599> the<00:00:13.759> exons<00:00:14.400> the<00:00:14.519> red

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blue and leave only the exons the red
 
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blue and leave only the exons the red
letters<00:00:15.559> generating<00:00:16.160> the<00:00:16.279> following<00:00:16.760> message

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letters generating the following message
 
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letters generating the following message
draw<00:00:17.640> a<00:00:17.840> big<00:00:18.119> house<00:00:18.439> on<00:00:18.600> the<00:00:18.720> corner<00:00:19.080> of<00:00:19.199> the

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draw a big house on the corner of the
 
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draw a big house on the corner of the
wide

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wide
 
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wide
Street<00:00:21.840> in<00:00:22.080> SMA<00:00:22.680> it<00:00:22.800> turns<00:00:23.119> out<00:00:23.439> that<00:00:23.599> during

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Street in SMA it turns out that during
 
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Street in SMA it turns out that during
RNA<00:00:24.480> splicing<00:00:25.400> the<00:00:25.599> seventh<00:00:26.039> Exxon<00:00:26.599> of<00:00:27.240> smn2

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RNA splicing the seventh Exxon of smn2
 
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RNA splicing the seventh Exxon of smn2
is<00:00:28.400> skipped<00:00:28.880> by<00:00:29.000> the<00:00:29.160> Machinery<00:00:29.679> that<00:00:30.000> edits

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is skipped by the Machinery that edits
 
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is skipped by the Machinery that edits
RNA<00:00:31.759> skipping<00:00:32.200> of<00:00:32.360> xon<00:00:32.759> 7<00:00:33.160> generates<00:00:33.640> a<00:00:33.760> faulty

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RNA skipping of xon 7 generates a faulty
 
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RNA skipping of xon 7 generates a faulty
message<00:00:35.000> draw<00:00:35.320> a<00:00:35.520> big<00:00:35.800> house<00:00:36.600> on<00:00:36.920> the<00:00:37.480> of<00:00:37.960> the

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message draw a big house on the of the
 
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message draw a big house on the of the
wide

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wide
 
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wide
Street<00:00:40.239> just<00:00:40.440> like<00:00:40.640> removing<00:00:41.079> word<00:00:41.360> number

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Street just like removing word number
 
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Street just like removing word number
seven<00:00:42.039> in<00:00:42.160> the<00:00:42.320> sentence<00:00:42.680> leads<00:00:42.960> to<00:00:43.160> a

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seven in the sentence leads to a
 
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seven in the sentence leads to a
statement<00:00:43.719> that<00:00:43.879> makes<00:00:44.160> no<00:00:44.399> sense<00:00:45.079> removing

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statement that makes no sense removing
 

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statement that makes no sense removing
exon7<00:00:46.480> in<00:00:46.640> the<00:00:46.760> RNA<00:00:47.320> results<00:00:47.760> in<00:00:47.920> a<00:00:48.079> defective

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exon7 in the RNA results in a defective
 
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exon7 in the RNA results in a defective
protein<00:00:50.239> remember<00:00:50.719> that<00:00:50.920> SMA<00:00:51.440> patients<00:00:51.879> have

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protein remember that SMA patients have
 
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protein remember that SMA patients have
to<00:00:52.199> rely<00:00:52.600> solely<00:00:53.440> on<00:00:53.600> the<00:00:53.760> sn2<00:00:54.559> gene<00:00:55.079> to

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to rely solely on the sn2 gene to
 
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to rely solely on the sn2 gene to
produce<00:00:55.680> the<00:00:55.960> survival<00:00:56.520> of<00:00:56.680> motor<00:00:57.039> neuron

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produce the survival of motor neuron
 
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produce the survival of motor neuron
protein<00:00:58.879> but<00:00:59.160> due<00:00:59.320> to<00:00:59.480> Exon<00:01:00.039> skipping<00:01:00.719> not

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protein but due to Exon skipping not
 
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protein but due to Exon skipping not
enough<00:01:01.559> usable<00:01:02.079> protein<00:01:02.600> is<00:01:02.760> made<00:01:03.000> to<00:01:03.199> support

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enough usable protein is made to support
 
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enough usable protein is made to support
normal<00:01:03.879> muscle development