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1 e and function (e.g., intron retention, exon skipping).
2 annot compensate for SMN1 loss due to exon 7 skipping.
3 n, and Sudemycins more acute effects on exon skipping.
4 on with these exonic sequences promotes exon skipping.
5 s to changes in stimulus duration) or period skipping.
6 current silent third base change, cause exon skipping.
7 lignancies to estimate the rate of said exon skipping.
8 ficient hDBR1 leads to a higher rate of exon skipping.
9 with different specificities to promote exon skipping.
10 sease course than those amenable to any exon skipping.
11 ic orientation that are favourable for water-skipping.
12 n to matched HC (n = 13) amenable to exon 51 skipping.
13 toring functional dystrophin protein by exon skipping.
14 und that the majority resulted in total exon skipping.
15 proteins are synthesized by way of ribosomal skipping.
16 g domain, with alternative splicing and exon skipping.
17 y mimic those that would be achieved by exon skipping.
18 lar domains and those that result in exon 14 skipping.
19 nscription rate causes an increase of exon 5 skipping.
20 and ssDNA gaps generated by replisome lesion skipping.
21 elayed neural tube closure, and altered exon skipping.
22 rnative splicing event, skipped exon or exon skipping.
23 rand gaps were generated by replisome lesion skipping.
24 gressed to the consecutive stage rather than skipping a stage.
25                 No similar functional prion, skipping a stop codon, has been found in Escherichia col
26 dical problem (OR, 3.0; 95% CI, 1.6 to 5.9); skipping a test, treatment, or follow-up (OR, 2.1; 95% C
27  > C), previously described to cause exon 10 skipping (a truncating alteration), displays characteris
28 ssociation study (GWAS) for breakfast cereal skipping, a commonly assessed correlated trait.
29 exons is widespread and correlates with exon skipping, a feature that adds considerably to the regula
30 tability of platelet RNA expression and exon skipping, a readily measured alternative splicing event.
31 , elastic spheres demonstrate superior water-skipping ability, but little is known about the effect o
32 istic explanation for the variations in exon-skipping activity and restoration of dystrophin protein
33  oligomers (PMOs) with glucose enhances exon-skipping activity in Duchenne muscular dystrophy (DMD) m
34 igher expression of hDBR1 only affected exon-skipping activity in malignant cells.
35 xpense of the inherent leading strand lesion skipping activity of the replisome, indicating that they
36                                         Exon skipping adds to the unexpected outcomes that must be ac
37 ted, c.[594-2A > C; 641A > G] caused exon 10 skipping, albeit not due to c.594-2A > C impairing the a
38 for two different Etaa1 mutations: an exon 2 skipping allele that deletes Gly78-Leu119, and a Cys166S
39       Antisense-oligonucleotide-induced exon skipping allows synthesis of partially functional dystro
40 ntly, a longer fasting period with breakfast skipping also increased the inflammatory potential of pe
41             Several agents targeting exon 14 skipping alterations are currently in clinical developme
42  across years was extremely high, even after skipping an entire seasonal migration.
43  studies revealed that it leads to mRNA exon skipping and ABCA4 protein truncation.
44 pre-mRNA is differentially spliced, via exon skipping and alternate 5'-splice donor sites, to yield f
45 ing in minigene assays, and resulted in exon skipping and an in-frame deletion of 40 amino acids in p
46  skipping as a proxy-phenotype for breakfast skipping and applied several in silico approaches to inv
47 periment to evaluate the effect of breakfast skipping and avoidance of ASF for 55 days.
48  nucleotide variant directly affects exon 14 skipping and changes the ratio of transmembrane versus s
49                   The mutation leads to exon skipping and deletion of 52-amino acid residues of a PX
50 splicing site mutations facilitating exon 16 skipping and deletions of exon 16 in a subset of these l
51  generates simultaneous expression of exon 6-skipping and G-nucleotide insertion variants.
52               The ASOs promote aberrant exon skipping and generation of premature termination codon (
53 nks between genetically determined breakfast skipping and higher body mass index, more depressive sym
54 I editing demonstrate that both the Alu exon skipping and inclusion isoforms encode active enzymes.
55  of novel transcriptomic events such as exon skipping and novel indels towards accurate downstream an
56 8-dependent association between SELP exon 14 skipping and race.
57 eotide conjugate designed to induce Dmd exon skipping and recover dystrophin protein expression cause
58 developmental stage, with activation of exon skipping and repression of intron retention being the mo
59 platelet splice QTL that alters SELP exon 14 skipping and soluble versus transmembrane P-selectin pro
60 from variable (V) Igkappa exons promote exon-skipping and synthesis of V domain-less kappa light chai
61                           Regulation of exon skipping and tandem alternative 3' splice sites (NAGNAGs
62  FLT3 resulted from complete or partial exon skipping and utilization of cryptic splice sites.
63 f MICA intron 4 leading to exon 3 and exon 4 skipping and/or deletions.
64 lls to characterize splicing variation (exon skipping) and its determinants.
65 ms included transcriptional repression, exon skipping, and intron inclusion.
66 correction of many genetic diseases via exon skipping, and the first AON-based drugs have entered cli
67 tation produce exon 33 skipping, exons 33-34 skipping, and WT VWF transcripts.
68 -term treatment with peptide-conjugated exon skipping antisense oligonucleotides (20-week regimen), r
69 ryonic dystrophin in zebrafish using an exon-skipping approach severely impairs the mobility and musc
70 pathology, compared to the single dystrophin skipping approach.
71 th other diseases that are eligible for exon-skipping approaches requiring whole-body treatment.
72 The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in
73 but alternative donor/acceptor site and exon skipping are mainly induced, indicating differential reg
74 ssing, alternative 5 and 3'ss usage and exon skipping are marked by distinct patterns of ordered intr
75                      As breakfast timing and skipping are not routinely measured in large cohort stud
76               These data highlight microexon skipping as a mechanism to spatially restrict signaling
77 genetic variants related to breakfast cereal skipping as a proxy-phenotype for breakfast skipping and
78 factor ETS variant 1 (ETV1) gene showed exon skipping at exon 7, while nudE neurodevelopment protein
79    While in most cases the Alu promotes exon skipping, at one locus the Alu increases exon inclusion.
80 ty, LaSSO identified both canonical and exon-skipping branch points.
81 hasing breakfast at a later time (P = 0.01), skipping breakfast (P = 0.03), and not preparing breakfa
82 ut downstream eating behaviors subsequent to skipping breakfast in free-living individuals.
83                                              Skipping breakfast is associated with an increased odds
84                                              Skipping breakfast is considered a frequent and unhealth
85 ompared with high-energy breakfast, habitual skipping breakfast was associated with a higher prevalen
86 sal link between being an evening person and skipping breakfast, but not vice versa.
87  health effects of specific eating patterns: skipping breakfast, intermittent fasting, meal frequency
88 y energy intake (70% of the population); and skipping breakfast, when consuming <5% of total daily en
89                                              Skipping breeding in female grey seals appears to be an
90                                              Skipping breeding increased subsequent pupping probabili
91                                         Exon skipping by ASOs is gaining traction as a therapeutic st
92 o protein encoding molecules, or induce exon skipping by disruption of exon splicing enhancers (ESEs)
93  alpha-chain mRNA splicing and favors exon 9 skipping by reducing the binding of the splicing enhance
94  We have now expanded the potential for exon skipping by testing whether an internal, in-frame trunca
95                These pathways include lesion skipping by the replisome, replication fork regression f
96 expressed by cancer cells with MSI, via exon skipping caused by somatic deletions in the T(17) intron
97 found that rs10401969 regulates SUGP1 exon 8 skipping, causing non-sense-mediated mRNA decay.
98 muscle disease and showed that multiple exon skipping could be induced in RNA that encodes a mutant h
99 ar trafficking correspond well with the exon-skipping data, with higher activity in myotubes than in
100 fat, and protein, respectively): a breakfast skipping day (BSD) and a dinner skipping day (DSD) separ
101  a breakfast skipping day (BSD) and a dinner skipping day (DSD) separated by a conventional 3-meal-st
102 , 24-h energy expenditure was higher on both skipping days (BSD: +41 kcal/d; DSD: +91 kcal/d; both P
103 s with deletions amenable to exons 44 and 46 skipping declined at a slower rate over 2 years (9 units
104 e muscular dystrophy, the rationale for exon skipping derived from observations in patients with natu
105 tely 80% cis), whereas species-specific exon skipping differences are driven by both cis- and trans-r
106 aucoma who reported taking smaller doses and skipping doses owing to cost dropped from 9.4% and 8.2%
107  if in the preceding 12 months they reported skipping doses to save money, taking less medication to
108 on because gene splicing analysis shows exon skipping due to loss of the ESE.
109 ircuit of sense-antisense pairs and the exon skipping during alternative splicing, through interactin
110  Mg(2+)-assisted reorientation, causing base-skipping during cDNA synthesis.
111                             We find that the skipping dynamics are governed by the wave propagation s
112 n normal adult tissues as a result of exon 6 skipping, enhanced exon 6 inclusion leads to expression
113                                         Exon skipping (ES) is reported to be the most common alternat
114                           Particularly, exon-skipping event in Enhancer of Zeste Homologue 2 (EZH2) a
115                           We present an exon skipping event in HDAC7, which is a candidate gene that
116 omplex PRC2, and the down-regulation of exon-skipping event may lead to the regain of functional EZH2
117           Both transcript isoforms from this skipping event site, i.e. in which the exon is either in
118 ing scanner (GESS), for de novo detection of skipping event sites from raw RNA-seq reads without prio
119 es with one single intron event and one exon skipping event were identified.
120             One particular mode of AS is the skipping event where an exon may be alternatively exclud
121 plice variants of KV10.1 resulting from exon-skipping events (E65 and E70) in human brain and cancer
122 ent between measured acceleration, number of skipping events and distanced traveled.
123 ew target function for AS prediction in exon skipping events and show it significantly improves model
124 atures due to ES events, and studies of exon skipping events associated with mutations and methylatio
125 identified by CoSREM may play a role in exon skipping events in several tumor samples.
126                                         Exon-skipping events increased in cells deficient for the nuc
127                       Interestingly, 29 exon-skipping events induced by treatment were identified and
128                                 Several exon skipping events were also highly repeatable, suggesting
129                      On the other hand, exon skipping events were rare in coding regions (1%) but enr
130 ll annotated introns, hundreds of novel exon-skipping events, and thousands of novel introns.
131 s, but also pinpointed novel, but rare, exon-skipping events, which may reflect aberrantly spliced tr
132 force algorithms to detect all possible exon-skipping events, which were widespread but rare compared
133 in pre-mRNA splicing with prevalence of exon skipping events.
134  to identify therapeutically targetable exon skipping events.
135 xon-intron boundary establishment leading to skipping events.
136 ith Becker MD and mdx mice subjected to exon skipping exhibited inhibited dystrophin degradation, pre
137 essor genes, including whole or partial exon skipping, exonification of intronic sequence, loss or ga
138 y to distinct KO strategies, for example, by skipping exons and reinitiating translation to potential
139 for the c.5842+1G>C mutation produce exon 33 skipping, exons 33-34 skipping, and WT VWF transcripts.
140 h an exon 5-targeted ASO-induced robust exon skipping for more than a year, improved motor coordinati
141 of CD46 cassette exon 13, whose inclusion or skipping generates different cytoplasmic tails with dist
142 aim of this study was to elucidate breakfast skipping genetic variants through a proxy-phenotype geno
143 idation of our approach in smaller breakfast skipping GWAS from the TwinUK (n = 2,006) and the Cohort
144 n of our signals in an independent breakfast skipping GWAS in another British cohort (P = 0.032), Twi
145 n, animal source food avoidanceand breakfast skipping has a significant desirable health effects on l
146                                         Meal skipping has become an increasing trend of the modern li
147 Indeed, antisense oligonucleotide-based exon skipping has shown promise for RDEB.
148 s.We investigated whether the timing of meal skipping impacts these risks by affecting circadian regu
149 nscripts by both directly increasing exon 13 skipping in an allele-related manner and specifically st
150 were shown to cause intron retention or exon skipping in an allele-specific manner, with approximatel
151                       ASOs also induced exon skipping in cell lines derived from patients with CLN3 B
152                    Given the success of exon skipping in clinical trials to treat genetic diseases su
153 fected AS events, with a preference for exon skipping in genes involved with cell mitosis.
154 idespread intron retention and cassette exon skipping in leukemic cells regardless of Srsf2 genotype,
155 t these CD33 SNPs are associated with exon 2 skipping in leukocytes from AML patients and with a nove
156 n by antisense oligonucleotide-mediated exon-skipping in mdx mice and (2) stable restoration of alpha
157 mous substitution that results in exon eight skipping in most mRNA molecules, ultimately leading to a
158 equence and is tightly regulated in favor of skipping in neurons; however, its biological function is
159 ce site recognition and contributing to exon skipping in nonneural cells.
160 : the novel PSEN1 variant resulted in exon 9 skipping in patient's RNA, leading to a pathogenic misse
161  to a silencer (ESS), causing frequent exon7 skipping in SMN2 pre-mRNA and yielding a truncated prote
162 n a reduction of dystrophin protein and exon skipping in the diaphragm.
163 binding site could efficiently induce exon 9 skipping in the presence of the G allele, and thereby re
164 soforms of Cask and Madd, and mimicking exon skipping in these transcripts through antisense oligonuc
165 hing and differential exon usage (i.e., exon-skipping), in addition to its effects on gene expression
166  involved in the regulation of HMGCR exon 13 skipping, in a founder population and in two distinct mi
167 ls after lunch.Compared with 3 meals/d, meal skipping increased energy expenditure.
168 herapeutic restoration of dystrophin by exon skipping induced widespread shifts in protein and mRNA e
169  can be placed into four major classes: exon skipping, intron retention, alternative 5' splice sites,
170 isense oligonucleotides (ASOs) mediated exon skipping is a promising therapeutic approach.
171                                         Exon skipping is a promising therapeutic strategy for Duchenn
172 mprove this therapeutic approach to DMD.Exon skipping is a strategy for the treatment of Duchenne mus
173                                         Exon skipping is a widespread phenomenon occurring across the
174 s study, by demonstrating that targeted exon skipping is able to rescue ciliary protein composition d
175                                         Exon skipping is considered a principal mechanism by which eu
176                                         Exon skipping is currently being tested in humans with dystro
177                           Although SMN2 exon skipping is the principal contributor to SMA severity, m
178 er included (inclusion isoform) or excluded (skipping isoform), are typically present in one cell, an
179 ted with a higher abundance of CYP2D6 exon 3 skipping junctions.
180 d that the c.893-1G>A mutation causes exon 7 skipping leading to deletion of 65bp, predicted to resul
181 (E12) as a model system because its in-frame skipping leads to a potentially functional protein.
182 ecific CPP-PMOs demonstrated comparable exon skipping levels for both pre-mRNA targets when compared
183  identified 6 genetic variants for breakfast skipping, linking clock regulation with food timing and
184  provided self-reported behaviors, including skipping meals and preparing meals at home.
185 r meals are difficult to distinguish because skipping meals and snacking have become more prevalent.
186                    We also described an exon-skipping mechanism for CARD11 dominant-negative activity
187 urrent in lung adenocarcinoma and cause exon skipping (METDelta14).
188                            To correct DMD by skipping mutant dystrophin exons in postnatal muscle tis
189 d family with a heterozygous in-frame exon 9 skipping mutation (c.835+2T>G) and in a CVID-affected fa
190 hogenic allele in exon 41, including an exon-skipping mutation that induced an in-frame deletion.
191 lear ribonucleoprotein H-dependent inclusion/skipping of a PRKCA alternative exon 3*.
192 ults in alternative splicing (AS), including skipping of an exon in amyotrophic lateral sclerosis (AL
193 e of PRRC2C, SRSF1 downregulation led to the skipping of an exon overexpressed in primary lung tumors
194 ly shown that these SNPs are associated with skipping of CD33 exon 2 in brain mRNA.
195      We show that the Alu insertion promotes skipping of CD58 exon 3 and results in a frameshifted tr
196 ice with morpholino oligomers to induce exon skipping of dystrophin exon 23 (that results in function
197 45 (Delta45)-may theoretically be treated by skipping of either flanking exon (44 or 46).
198 difications, but also before them, through a skipping of either some or all of these modifications wi
199 that results in a splicing defect leading to skipping of exon 11 (p.Lys333Lysfs*3).
200                    In the absence of ELAVL1, skipping of exon 11 of Eif4enif1 forms the stable, short
201 the frameshift p.Lys645AlafsTer12 and/or the skipping of exon 11 that results in deletion of 30 highl
202 erited a splice-site variant that results in skipping of exon 13, p.Ile459Profs( *)4 (not reported in
203 cancer, alternative splicing of ERBB2 causes skipping of exon 16, leading to the expression of an onc
204 th endplate AChE deficiency causes exclusive skipping of exon 16.
205 KBKAP gene that results in a tissue-specific skipping of exon 20 and a corresponding reduction of the
206  as IKBKAP); this mutation leads to variable skipping of exon 20 and to a drastic reduction of ELP1 i
207 o additional novel transcripts involving the skipping of exon 3 or exons 3, 4, and 5 and are referred
208                  Patient-derived PPCs showed skipping of exon 39 or exons 39 and 40 in the mRNA.
209 uman NRP1 splice variants resulting from the skipping of exon 4 and 5, respectively, in colorectal ca
210  conclude that in DMD patients with Delta45, skipping of exon 44 and multiexon skipping of exons 46 a
211 s 46-48) are better potential therapies than skipping of exon 46 alone.
212 to those of full-length channels (Cav1.4FL), skipping of exon 47 dramatically alters Cav1.4 function.
213 ense oligonucleotide-mediated (ASO-mediated) skipping of exon 6 decreased MDM4 abundance, inhibited m
214 a duplicate gene (SMN2) primarily results in skipping of exon 7 and production of an unstable protein
215 ompensate for this deficiency because of the skipping of exon 7 during pre-messenger RNA (mRNA) proce
216 ls to compensate for the loss of SMN1 due to skipping of exon 7.
217 h causes premature protein truncation due to skipping of exon 7.
218 site mutation (c.730+4A>G), causing in-frame skipping of exon 8.
219 l fibroblasts showed splice-variant mediated skipping of exon 9 leading to an out-of-frame product an
220 HHt-causing CUL3 mutations, resulting in the skipping of exon 9, lead to a more severe phenotype.
221 h Delta45, skipping of exon 44 and multiexon skipping of exons 46 and 47 (or exons 46-48) are better
222 g cDNA showed the splice site variant led to skipping of exons 6 of the FHL1A isoform, equivalent to
223                                              Skipping of exons 7 and 8 is predicted to delete critica
224 e variant, P2RX7L, that is, characterized by skipping of exons 7 and 8.
225 nne muscular dystrophy, we propose that exon skipping of FcepsilonRIbeta is a potential approach for
226 re-mRNA, is necessary and sufficient for the skipping of L1CAM transmembrane domain in ECs, leading t
227 er to detect the EGFP fluorescence only when skipping of mdx-type exon 23 is induced by ASOs.
228                    Differential inclusion or skipping of microexons is an increasingly recognized cla
229                   Consistently, we show that skipping of MKK7 exon 2 enhances JNK pathway activity, a
230 disrupted the developmentally regulated exon skipping of Ndel1 mRNA, which is bound by MBNL1 and crit
231 scination and engineering application, water-skipping of rigid stones and spheres has received consid
232       One of the most repeatable was exon 14 skipping of SELP.
233  calcium levels induce robust and reversible skipping of several alternative exons from endogenously
234 euron 1 (SMN1) gene coupled with predominant skipping of SMN2 exon 7.
235 antisense oligonucleotides (AONs) to promote skipping of specific exons such that the reading frame i
236 sing antisense oligonucleotide-mediated exon skipping of the beta-subunit of the high-affinity IgE re
237 to N-terminally truncated target proteins or skipping of the edited exon leading to protein isoforms
238 in a PTBP1-binding site that disrupts normal skipping of the FLNA poison exon in NPCs and causes a br
239 es an autoinhibitory domain, induces partial skipping of the in-frame exon and nuclear accumulation o
240                                     Targeted skipping of the mutated exon c.5668 G > T using antisens
241 al-wintering sanderlings, in addition to the skipping of this area during northward but not southward
242 se oligonucleotides to reverse the increased skipping of TRA2beta-PE detected in breast tumors, alter
243 on induced by histone depletion promotes the skipping of variable exons in the CD44 gene.
244                              Here using exon skipping oligonucleotides we predominantly restored dyst
245 gate the effect of ASF fasting and breakfast skipping on micronutrient intake and determine the minim
246 oidance of animal source foods and breakfast skipping) on lipid profiles, fasting blood sugar and bod
247 near the 3' splice site promoted either exon skipping or inclusion.
248 e single-guide RNAs (sgRNAs) can induce exon skipping or large genomic deletions that delete exons.
249 uencing regulatory elements, leading to exon skipping, or by creating a new cryptic splice site.
250 At low temperatures, we observed caustics of skipping orbits extending over hundreds of superlattice
251 ation dynamics, we further observed both the skipping orbits of excited atoms traveling down the syst
252 nuniform sampling (NUS) obtained by randomly skipping points in the first dimension corresponding to
253 on through micro-dystrophin delivery or exon skipping, preclinical models have shown that incomplete
254 n vitro that the GH1 variant leads to exon 3 skipping, predicting a mutant protein known to cause hum
255 we built the winning model of the CAGI5 exon skipping prediction challenge.
256                            Drug-induced exon skipping preferentially affects shorter alternatively sp
257 cing modulator-induced intron-retention/exon-skipping profile, which correlates with the differential
258 sion synthesis came at the expense of lesion-skipping replication restart.
259                             AON-induced exon skipping resulted in specific Alk4 down-regulation, inhi
260 er confirmed that all 3 variants caused exon skipping resulting in frameshifts that lead to premature
261 novel computational method, graph-based exon-skipping scanner (GESS), for de novo detection of skippi
262 l not capture the fitness benefits gained by skipping sexual reproduction.
263 tations at splice junctions often cause exon skipping, short deletions, or insertions in the mature m
264   The subset of patients amenable to exon 51 skipping showed a more severe disease course than those
265                                The option of skipping similarity matching is also available so that t
266 sortium and experimentally validated several skipping site predictions by RT-PCR.
267 bined to predict effects of variants on exon skipping, splice site choice, splicing efficiency, and p
268 s and increased fat oxidation with breakfast skipping suggest the development of metabolic inflexibil
269  in patients with mutations amenable to exon skipping than in those not amenable.
270 role is to funnel a rough folding landscape, skipping the accumulation of kinetic traps, providing a
271 ing with a new partner would be paid through skipping the subsequent breeding attempt.
272  three families and was shown to induce exon skipping; the other mutations were frameshift mutations
273                                         Exon-skipping therapies aim to convert Duchenne muscular dyst
274 eletions and insertions are targeted by exon-skipping therapies.
275 mice, combined dystrophin and myostatin exon skipping therapy greatly improved DMD pathology, compare
276 mers (PMOs) has shown great promise for exon-skipping therapy of Duchenne Muscular Dystrophy (DMD).
277 deno-associated virus (AAV)-U7-mediated exon-skipping therapy was shown to decrease drastically after
278 implications for the development of DMD exon-skipping therapy.
279 well as the biodistribution of ASOs for exon skipping therapy.
280   Although very effective in correcting exon skipping, they also induced retention of the short downs
281 ding nonsense variants, induced in-frame E12 skipping through the modification of splice sites or reg
282 also affects splicing by facilitating exon 2 skipping, thus also being in effect a loss-of-function a
283 ense oligonucleotide (ASO) that induces exon skipping to restore the open reading frame.
284 lement, the subjects completed 6 min of rope-skipping to stimulate collagen synthesis.
285 toration of wt dystrophin levels, using exon-skipping, together with increased utrophin levels restor
286                                   These exon-skipping transcripts are therefore unlikely to be functi
287                           The pervasive exon-skipping transcripts were stochastic, did not increase i
288 pe 1 VWD through mRNA degradation of exon 38 skipping transcripts.
289                                         Exon skipping uses antisense oligonucleotides (ASOs) to alter
290                                         Exon skipping uses antisense oligonucleotides as a treatment
291  corresponding to LBD to produce namely exon-skipping variants.
292            The average splicing rate by exon skipping was approximately 0.24% in wild type and approx
293 ly from on-target integration, and ribosomal skipping was highly efficient.
294         The experiment reveals a new mode of skipping, "water walking", which is observed for relativ
295                              To prevent exon-skipping, we have targeted an intronic repressor, Elemen
296 mitant with T cell activation-induced exon 5 skipping, we observed a downregulation of SRSF1.
297  polymerase chain reaction to detect exon 14 skipping were performed where sufficient tissue was avai
298  were >/=7 years old and amenable to exon 51 skipping were randomized to eteplirsen (30/50mg/kg) or p
299 regulated exons PTBP1 tends to promote their skipping, whereas binding to downstream sites often stim
300 d reading frame upstream of the IRES by exon skipping, which led to synthesis of a functional N-trunc

 
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