ライフサイエンスコーパス: gene mutation

1 creased aortic risk in patients with an FBN1 gene mutation.

2 ses revealed distinct profiles for each PRNP gene mutation.

3 n individuals who bear the same germline NF1 gene mutation.

4 icroraft array and screened for the targeted gene mutation.

5 andidate genes were validated by independent gene mutation.

6 epigenetic regulators, and tumor suppressor gene mutation.

7 disease ascribed solely to single sarcomere gene mutations.

8 ity of CHD cases can be attributed to single gene mutations.

9 ors in children and adolescents carrying few gene mutations.

10 associated with cationic trypsinogen (PRSS1) gene mutations.

11 minant families carried pathogenic Mendelian gene mutations.

12 associated with potentially harmful BRCA1/2 gene mutations.

13 ition to cancer in carriers of some of these gene mutations.

14 presence of two or more high molecular risk gene mutations.

15 associated with potentially harmful BRCA1/2 gene mutations.

16 fast, and can identify both known and novel gene mutations.

17 lective expansion of HSPCs carrying specific gene mutations.

18 ly 50% of peripheral blood CLL cases lacking gene mutations.

19 patients with early-onset CRC, 72 (16%) had gene mutations.

20 nd FLT3-internal tandem duplication and NPM1 gene mutations.

21 ) is a progeroid-like syndrome caused by WRN gene mutations.

22 ts were detected to carry heterozygous TGFBI gene mutations.

23 ates the landscape of potentially targetable gene mutations.

24 nsequences of naturally occurring regulatory gene mutations.

25 "at risk" patients with identified Gly203Ser gene mutations.

26 D subjects and 1,789 controls for connectome gene mutations.

27 urative) for 190 gene expressions and for 31 gene mutations.

28 reflect the cumulative effect of individual gene mutations.

29 n induced by nephritic factors or complement gene mutations.

30 ation fork stability, potentially leading to gene mutations.

31 elial carcinomas, tumors associated with TSC gene mutations.

32 er (ASD) is strongly associated with de novo gene mutations.

33 articipants had moderate-penetrance CRC risk gene mutations (19 monoallelic MUTYH, 17 APC*I1307K, two

34 Of the 1437 patients with an FBN1 gene mutation, 26 patients (1.8%) had a BAV.

35 mors, and 40 patients (83.3%) had at least 1 gene mutation: 37 had Lynch syndrome (13, MLH1 [includin

36 tumors, and 32 patients (8%) had at least 1 gene mutation: 9 had mutations in high-penetrance CRC ge

37 ncluding the presence of TP53 or spliceosome gene mutations, a variant allele fraction >10%, the pres

38 Cardiac troponin I (TNNI3) gene mutations account for 3% of hypertrophic cardiomyop

39 We report that major truncating BCOR gene mutation affecting HSPC and CMP was beneath the thr

40 However, the neural mechanism whereby Shank3 gene mutations affects cortical functional connectivity

41 Specific gene mutations also had an impact on the outcome of the

42 nts, selective pressure in vivo resulting in gene mutations, altered expression of redox-active prote

43 primary structure alterations resulting from gene mutations, alternative splicing, post-translational

44 The uniformity of known driver gene mutations among metastases in the same patient has

45 quency and spectrum of cancer susceptibility gene mutations among patients with early-onset CRC.

46 or quantitative protein detection as well as gene mutation analysis with applications in next-generat

47 nia patients with epsilon-sarcoglycan (SGCE) gene mutation and 21 age- and sex-matched healthy contro

48 uillarum 531 Ac and 531Ad differ in the hmgA gene mutation and 23 mutations, most of which locate to

49 Because of the highly penetrant gene mutation and clinical features consistent with idio

50 Notably, the ALKBH5 gene mutation and expression status of melanoma patients

51 tments cells and systems make in response to gene mutation and help to identify mechanisms conferring

52 rofile of MTC with regard to the type of RET gene mutation and the cancer genetic background (heredit

53 insight into recently identified complement gene mutations and brain disorders.SIGNIFICANCE STATEMEN

54 nal burden downstream of DNA mismatch repair gene mutations and composite gene expression score-based

55 yproduct of evolutionary mechanisms, such as gene mutations and epigenetic modifications, which is ma

56 d to update surgeons and academics on driver gene mutations and epigenetics in colorectal cancer.

57 studies point to novel associations between gene mutations and immune surveillance that could impact

58 istic qAF levels in association with certain gene mutations and in participants without detected muta

59 o previous studies of ccRCC, in pRCC, driver gene mutations and most arm-level somatic copy number al

60 nt to the important association of filaggrin gene mutations and other skin barrier defects with AD.

61 P1 is a downstream phenotypic commonality of gene mutations and predicts outcome following rituximab-

62 3, and other (pfdhfr, pfdhps, pfmdr1, pfcrt) gene mutations and survival of parasites as detected by

63 Here, to identify genes, mutations and biological processes that give sele

64 or aberrant expression in the protein-coding genes, mutations and misregulation of noncoding RNAs, in

65 (0/15), 19% (5/27) failure in cases with one gene mutation, and 69% (11/16) failure in cases with mor

66 ereditary pancreatitis, patients with CDKN2A gene mutation, and patients with 1 or more first-degree

67 Vfs*41 and its association with other cancer gene mutations, and found that the mutation occurred nea

68 ied several somatic copy-number alterations, gene mutations, and the basal expression of gene sets th

69 omogeneous with respect to functional driver-gene mutations, and we suggest that future efforts to de

70 Lamin A/C (LMNA) gene mutations are a known cause of familial dilated car

71 K13 gene mutations are a primary marker of artemisinin resis

72 These results establish that driver gene mutations are associated with methylation alteratio

73 nction and mechanisms of action, except that gene mutations are associated with risks of primary macr

74 n NADPH-cytochrome P450 oxidoreductase (POR) gene mutations are associated with severe skeletal defor

75 e clinical observation that rare coding GLRB gene mutations are associated with the neurological diso

76 Conclusion Germline cancer susceptibility gene mutations are carried by 9.9% of patients with CRC.

77 Although most common laryngeal gene mutations are found in both subclasses, better prog

78 Somatic gene mutations are key determinants of outcome in patien

79 to recapitulate the CMML phenotype, and many gene mutations are loss of function, making the identifi

80 n the case of cancer samples combinations of gene mutations are related to patterns of gene expressio

81 Cooperating gene mutations are typically required to transform norma

82 ng from patched domain containing 1 (Ptchd1) gene mutations are unknown.

83 ins of A. fumigatus with a variety of cyp51A gene mutations, as well as the triazole-resistant strain

84 Phosphatidylinositol glycan class A (PIG-A) gene mutation assay phenotypically measures erythrocyte

85 equency as detected by thymidine kinase (TK) gene mutation assay.

86 teral sclerosis (ALS) is whether the various gene mutations associated with the disease converge on a

87 The driver gene mutation-associated methylation differences between

88 Herein, we show a new strategy for the gene mutation (BRAF c.1799T>A; p.

89 The acquired gene mutations, broad genomic alterations, and gene expr

90 e confirmed this heterogeneity and show many gene mutations but without a unifying mutation.

91 alysis of 67 tumors and validation of driver gene mutations by exome sequencing of 45 tumors.

92 We identified a family with a UMOD gene mutation (C106F) resulting in glomerular inflammati

93 Somatic gene mutations can alter the vulnerability of cancer cel

94 Nonsynonymous gene mutations can be beneficial, neutral, or detrimenta

95 Complement gene mutations can cause familial C3 glomerulopathy, and

96 Recent studies have shown that gene mutations can serve as prognostic and/or predictive

97 the TRACK-HD cohort of Huntington's disease gene mutation carriers (data collected 2008-11).

98 ic Cardiomyopathy) targeted young sarcomeric gene mutation carriers with early-stage hypertrophic car

99 l phrase "to identify carriers of first-wave gene mutation carriers" should have instead read "to ide

100 Biallelic CD19 gene mutations cause common variable immunodeficiency in

101 insight into the mechanisms behind how titin gene mutations cause hereditary cardiomyopathy and how t

102 Several P2 gene mutations cause human Charcot-Marie-Tooth neuropath

103 TRIM37 gene mutations cause muscle-liver-brain-eye (mulibrey) n

104 Glutathione metabolism gene mutations caused regionally distinct changes in the

105 KRAS gene mutation causes lung adenocarcinoma.

106 With a spectrum of podocyte-expressed gene mutations causing chronic disease, an enhanced unde

107 Gene mutations causing complete loss of function account

108 demyelinating neuropathy, results from GJB1 gene mutations causing loss of function of the gap junct

109 dentified the genetic background behind rRNA gene mutations causing variable levels of resistance to

110 an exponential rate, yet characterizing the gene-mutation-cell-behavior relationships essential for

111 cluding survivors with cancer predisposition gene mutations, chest radiation 10 Gy or greater, or bot

112 ic neurons from patients with parkin (PARK2) gene mutations compared to those from healthy controls.

113 Recessive LRPAP1 gene mutations confer a high risk of childhood-onset RRD

114 TATEMENT Genetic studies indicate that TREM2 gene mutations confer increased Alzheimer's disease (AD)

115 mage and after zinc finger nuclease-mediated gene mutation correction, mtDNA damage was no longer det

116 n in the interpretation of ribosomal protein gene mutation data.

117 -increasing mutations in MC4R from the Human Gene Mutation Database (HGMD) and Clinvar.

118 single nucleotide variants (SNVs) from Human Gene Mutation Database and 10 002 putatively 'benign' no

119 set of human variants derived from the Human Gene Mutation Database, ClinVar and the Exome Aggregatio

120 arger collection of mutations from the Human Gene Mutation Database, MAPPIN is able to significantly

121 ntially by germline variation from the Human Gene Mutation Database, recurrent somatic variation from

122 of encoded proteins was examined using human gene mutation databases.

123 Furthermore, all functional driver-gene mutations detected in these 14 primary tumours were

124 d the highest diagnostic performance for IDH gene mutation detection in low-grade glioma (AUC, 0.818)

125 The distribution of CHIP-related gene mutations differs between individuals with therapy-

126 stream, highly interconnected regulatory ASD gene mutations disrupt transcriptional programs or signa

127 d reveal mechanistic distinctions between RP gene mutations driving hematopoietic disease and those r

128 a baseline for understanding the effects of gene mutations during development, as well as a roadmap

129 Cancer gene mutations (e.g., KRAS, TP53) and microsatellite sta

130 own as the p.K210del) and the non-sarcomeric gene mutation encoding lamin A/C (LMNAp.R331Q ).

131 ase (FD) caused by GLA(alpha-galactosidase A gene) mutations encoding p.D322E (family A) or p.I232T (

132 All patients with an FBN1 gene mutation evaluated in our clinic were included.

133 Specifically, clock gene mutations, exposure to artificial light-dark cycles

134 However, most murine models based on single gene mutations fail to recapitulate the CMML phenotype,

135 Causative gene mutations (for example, NOTCH1, SMAD6) are known fo

136 eloping schwannomas with the same underlying gene mutations found in schwannomatosis patients.

137 These findings support that gene mutations found on single pretreatment biopsies wil

138 nd beta-esterase activities, but not in Vgsc gene mutation frequency, suggesting metabolic detoxifica

139 These methods have also been used to predict gene mutations from pathology images, but no comprehensi

140 tion process, fukutin related protein (FKRP) gene mutations generate a wide range of pathologies from

141 Thus, the ENO gene mutation gives rise to plants that yield larger mul

142 All but one tumour have a known UM driver gene mutation (GNAQ, GNA11, BAP1, PLCB4, CYSLTR2, SF3B1,

143 Patients who carry the BRCA1 and BRCA2 gene mutations have an underlying genetic predisposition

144 However, no CYP2B11 gene mutations have been identified that explain this de

145 ypothesize that cells harbouring spliceosome gene mutations have increased sensitivity to pharmacolog

146 ignificantly, circadian and clock-controlled gene mutations have recently been identified by Genome-W

147 Altered bile acid metabolism resulting from gene mutations, high-fat diets, alcohol, or circadian di

148 ), and having a breast cancer predisposition gene mutation (HR, 23.0; 95% CI, 7.3 to 72.2).

149 en the margin and original IPMN using driver gene mutations identified by next-generation sequencing.

150 0(9)/L), presence of one high molecular risk gene mutation (ie, in SRSF2, ASXL1, and/or RUNX1), and p

151 We performed (i) meta-analysis for each gene mutation; (ii) weighted ordinary linear regression

152 BRCA1 gene mutations impair homologous recombination (HR) DNA

153 e CRISPR/Cas9 to generate the first targeted gene mutation in A. burtoni and show that Ptgfr is neces

154 fragment of human mutant HTT, the causative gene mutation in Huntington's disease.

155 is neuronal 3), which is modified because of gene mutation in juvenile neuronal ceroid lipofuscinosis

156 Because of a gene mutation in the FECH gene, ferrochelatase, the enzy

157 he successful correction of a cardiomyopathy gene mutation in viable human embryos.

158 tumours with multiple samples, 97% of driver-gene mutations in 38 patients were homogeneous.

159 tion status, common genomic aberrations, and gene mutations in 421 untreated patients within the CLL1

160 ve disorder caused by fully penetrant single gene mutations in a minority of cases, while the majorit

161 Single gene mutations in at least 50 genes have been proposed t

162 The prevalence of driver gene mutations in carcinomas with the intestinal phenoty

163 Inherited gene mutations in cholesterol metabolism result in a sev

164 Different gene mutations in endometrial cancer cells have varied r

165 In this study, we analyzed the RP2 and RPGR gene mutations in five Han Chinese families with XLRP.

166 Thus, we propose that discrete gene mutations in intellectual disability can generate "

167 Thus, we propose that discrete gene mutations in intellectual disability might generate

168 redox state using drug strategies and single-gene mutations in isocitrate dehydrogenases (IDH1/2).

169 Gene mutations in L-type voltage gated calcium channel s

170 metabolites, which are produced by metabolic gene mutations in many cancers, sensitize cells to PARP

171 Single-gene mutations in patients can help define genetic facto

172 prevalence of germline cancer susceptibility gene mutations in patients with CRC unselected for high-

173 his setting in view of the high frequency of gene mutations in pediatric cerebrospinal AVFs, and show

174 the multiple variants of TMPRSS2:ERG fusion gene mutations in prostate cancer (PCa), are promising d

175 ion of dominantly inherited alpha-syn (SNCA) gene mutations in rare cases of familial PD.

176 Gene mutations in the chromatin remodeling groups were r

177 , hereditary diseases associated with single gene mutations in the Kennedy pathways have been identif

178 hormonal, and structural effects of CYP11B1 gene mutations in the largest international cohort of 10

179 n familial ALS phenotypes linked to multiple gene mutations including the TDP-43 gene coding (TARDBP)

180 (7.0%) of 1,058 participants carried non-LS gene mutations, including 23 (2.2%) with mutations in hi

181 cancers are associated with several critical gene mutations, including PIK3A, PTEN, and KRAS.

182 foster tumorigenesis rather than new driver gene mutations, including significant pancreatic islet c

183 method for generating cells carrying precise gene mutations, including the rearrangement and deletion

184 Our study shows that different gene mutations induce DCM via diverse cellular pathways.

185 Our study shows that different gene mutations induce dilated cardiomyopathy via diverse

186 There are many disorders, and causes include gene mutations, infections, or exposure to toxins.

187 hese studies establish that the germline Nf1 gene mutation is a major determinant of optic glioma dev

188 A GNB2 gene mutation is associated with familial SND+AVB and le

189 rrier probabilities of cancer susceptibility gene mutations is an important part of pre-test genetic

190 pathy (HCM) caused by sarcomere protein (SP) gene mutations is current standard of care, but there ar

191 creased inflammasome activity resulting from gene mutations is related to monogenic autoinflammatory

192 We found that Klotho gene mutation (KL-/-) largely decreased serum klotho lev

193 ents in the skin (FLG and other skin barrier gene mutations, Langerhans cells, type 2 innate lymphoid

194 PI3K-delta hyperactivity resulting from the gene mutations leads to similar clinical presentations,

195 tablished iPSC lines that contain sarcomeric gene mutations linked to cardiomyopathy in patient popul

196 t research suggests that DNA mismatch repair gene mutations may facilitate acquisition of resistance

197 earrangements had a low number of additional gene mutations (median, 1; range 0 to 6), which involved

198 ems because of the lack of comparable single gene mutation models.SIGNIFICANCE STATEMENT All sensory

199 Ribosomal protein (RP) gene mutations, mostly associated with inherited or acqu

200 nrecognized pancreatic cancer susceptibility gene mutation (nine that involved ATM, two BRCA2, one BR

201 These cells, in the absence of any driver gene mutations, now transform by introducing a single KR

202 tary autoinflammatory diseases are caused by gene mutations of the innate immune pathway, e.g. nucleo

203 The frequency of gene mutations of the pathway was significantly higher i

204 Furthermore, somatic gene mutations of varied functional classes license the

205 genes are expressed as well as the impact of gene mutation on function throughout life, in tandem wit

206 renewed approach to calculate the impact of gene mutations on the binding affinity through the struc

207 findings, studies to determine the impact of gene mutations on the trafficking of the Caenorhabditis

208 kott-Aldrich syndrome, defined by either WAS gene mutation or absent Wiskott-Aldrich syndrome protein

209 lts suggest functional disruption of M30 via gene mutation or altered expression as a convergent mech

210 ulation of misfolded proteins as a result of gene mutations or abnormal protein homeostasis.

211 PIK3CA gene mutations or amplifications that affect the PI3K p1

212 s9 can be applied to correct disease-causing gene mutations or engineer T cells for cancer immunother

213 lar alterations (IDH2 mutations, spliceosome gene mutations) or altered signaling pathways (BCL2 inhi

214 rage, and genetic analysis confirmed a GLA A gene mutation (p.Asn215Ser) in all 3 patients.

215 Here we report a pneumococcal pbp1b gene mutation (pbp1bA641C causing N214T change in PBP1b

216 Most gene mutations persisted in CR, albeit at highly variabl

217 e systems.Mounting evidence show that driver gene mutations play only part of the role in carcinogene

218 cent studies show that aneuploidy and driver gene mutations precede cancer diagnosis by many years(1-

219 The extent of heterogeneity among driver gene mutations present in naturally occurring metastases

220 o applied to analyze the genome-wide somatic gene mutation rate difference between lung adenocarcinom

221 vestigate the biological role of these RSV G gene mutations, recombinant RSV strains harboring either

222 itrate dehydrogenase 1 (IDH1) and IDH2 (28%) gene mutations, recurrent arm-length copy number alterat

223 Neonatal diabetes is caused by single gene mutations reducing pancreatic beta cell number or i

224 s disease (HD) is caused by Huntingtin (Htt) gene mutation resulting in the loss of striatal GABAergi

225 B-thalassemia is caused by B-globin gene mutations resulting in reduced (beta(+)) or absent

226 Objective: To identify the gene mutation(s) accountable for the mottled pigmentatio

227 Finally, diffusing pan-cancer gene mutation scores based on different Graphlet Laplaci

228 GD patients with genetically confirmed ABCA4 gene mutations seen at the Wilmer Eye Institute with fol

229 inal center B-cell (GCB) subtype and carried gene mutations similar to the adult counterpart (eg, SOC

230 We also noticed that the site of the RET gene mutation slightly influenced the gene expression pr

231 deleterious pancreatic cancer susceptibility gene mutations, some of which are therapeutically target

232 t strand bias, to enlarge the immunoglobulin gene mutation spectrum from G-C to A-T bases.

233 terization of isocitrate dehydrogenase (IDH) gene mutation status of gliomas.

234 s, including laterality, family history, and gene mutation status were analyzed.

235 tratified by homologous recombination repair gene mutation status, progression-free interval after th

236 ion based on homologous recombination repair gene mutation status, progression-free interval followin

237 performance for grading and detection of IDH gene mutation status.

238 and progression, despite a common underlying gene mutation, strongly suggesting the involvement of ot

239 or FLT3-internal tandem duplication and NPM1 gene mutation subgroups.

240 CAR-T cells targeting tumour-specific driver gene mutations, such as the four-nucleotide duplication

241 able penetrance among patients with the same gene mutations suggests involvement of additional mechan

242 es in rin/rin mutant, which harbors MADS-RIN gene mutation, suggests that MADS-RIN transcription fact

243 he likelihood of missing a functional driver-gene mutation that was present in all metastases was 2.6

244 with NADH dehydrogenase subunits and nuclear gene mutations that affect mitochondrial function result

245 te (MK) and thrombopoiesis in the context of gene mutations that affect sialylation and galactosylati

246 onal somatic karyotypic abnormalities and/or gene mutations that aid in the diagnosis and can be used

247 These data suggest that single gene mutations that cause neurological diseases such as

248 ontrolled by genetics as shown by the single-gene mutations that confer extreme early or late chronot

249 en developed to detect the 23S ribosomal RNA gene mutations that confer resistance to azithromycin.

250 sed the 2009 pandemic, it evolved polymerase gene mutations that enabled it to more efficiently use h

251 y cases, this is the consequence of specific gene mutations that have the potential to be targeted to

252 w failure syndrome associated with ribosomal gene mutations that lead to ribosomal insufficiency.

253 Inherited hearing loss is associated with gene mutations that result in sensory hair cell (HC) mal

254 A total of 84 germline DNA-repair gene mutations that were presumed to be deleterious were

255 Initial or truncal TP53 gene mutations (the first mutations in a stem cell) are

256 typic variability associated with regulatory gene mutations, the rapid examination of these genes in

257 ead read "to identify carriers of first-wave gene mutation." The error has been corrected in the HTML

258 who have an ancestry associated with BRCA1/2 gene mutations, there is adequate evidence that the bene

259 ausible pathophysiologic mechanism for Scn1a gene mutation to impair spatial memory.

260 izations (USVs) are a vital tool for linking gene mutations to behavior in mouse models of communicat

261 from genetic polymorphisms and cancer-driver gene mutations to obesity, chronic inflammation, and imm

262 in conjunction with diabetes, hypertension, gene mutations, toxins or infections but may also be of

263 al of less fit cells ('losers') but why some gene mutations turn cells into losers is unclear.

264 the correlation between mild and severe CFTR gene mutation types and lipid profiles, suggesting a pos

265 This study investigated the TGFBI gene mutation types in outpatients clinically diagnosed

266 indings challenge the paradigm of complement gene mutations typically causing primary membranoprolife

267 Homozygous or compound heterozygous IL36RN gene mutations underlie the pathogenesis of psoriasis-re

268 L, and our findings narrow the repertoire of gene mutations useful in minimal residual disease-based

269 ential connections for anti-cancer drugs and gene mutations using DI in pharmacogenomic data.

270 Thyroid hormone receptor alpha (THRA) gene mutations, via dominant negative mode, cause erythr

271 patients (2%) with the CT genotype, a SPINK1 gene mutation was found, while in the control group it w

272 No EphB4 gene mutation was identified.

273 The PLG gene mutation was present in all studied symptomatic pat

274 The presence of sarcomeric gene mutations was associated with increased occurrence

275 3 signature mutations, the frequency of tRNA gene mutations was much higher than in the rest of the g

276 e (MRD) status, genomic complexity (GC), and gene mutations were assessed.

277 DNA damage response gene mutations were associated with higher TMB (P < 0.00

278 Notably, gene mutations were detected in all the major AML-associ

279 Gene mutations were determined by polymerase chain react

280 50 years were included in the study, and 75 gene mutations were found in 72 patients (16%).

281 MEN1 gene mutations were found to be exclusively enriched in

282 ss the tested CF population as a whole, CFTR gene mutations were found to be the primary determinant

283 erent disease-associated loci and pathogenic gene mutations were identified in each family, including

284 r (NR) genes, genetic disorders caused by NR gene mutations were initially discovered by a candidate

285 Histone gene mutations were more frequent than previously report

286 -containing E3 ubiquitin ligases, and KLHL15 gene mutations were recently described as a cause of sev

287 Specific gene mutations were too infrequent in patients with spec

288 The most frequent somatic gene mutations were TP53 (65.6%), followed by KRAS (48.1

289 cancer cell lines with PIK3A, PTEN, and KRAS gene mutations were treated with docetaxel and radiation

290 e genetics of drug resistance, novel somatic gene mutations which allow a tumour to evade therapeutic

291 e a common cell of origin and similar driver-gene mutations which divert cell fate from differentiati

292 Passenger gene mutations, which do not have known or predicted fun

293 ma were screened for NF2, SMARCB1, and LZTR1 gene mutations, while patients with meningioma were scre

294 opportunity for targeting this common driver gene mutation with antibody-based therapies.

295 who have an ancestry associated with BRCA1/2 gene mutations with an appropriate brief familial risk a

296 A prediction is that ion channel gene mutations with equivalent effects on neuronal excit

297 The integration of somatic gene mutations with gene expression signatures provides

298 rognosis in adults who may also harbor other gene mutations with oncogenic potential as they age.

299 e been described, many of which carry single-gene mutations within the growth-hormone, insulin/IGF-1

300 ate for the lack of dystrophin caused by DMD gene mutations, without the immunogenic concerns associa