ライフサイエンスコーパス: gene therapy

1 herapeutic modality (microbiome-biosynthetic gene therapy).

2 nsplantation and vectors for the delivery of gene therapy.

3 A total of 17 boys received Lenti-D gene therapy.

4 nt implications for using minicircle DNA for gene therapy.

5 yet to overcome for HAdV-5-mediated systemic gene therapy.

6 pment of HAdV-5-based adenoviral vectors for gene therapy.

7 s in the cerebrospinal fluid 12 months after gene therapy.

8 ional genomic research and holds promise for gene therapy.

9 Foamy virus is an attractive vector for gene therapy.

10 transduction enhancers for potential use in gene therapy.

11 task in molecular biology, biotechnology and gene therapy.

12 evade NAbs in prospective patients for human gene therapy.

13 e can eliminate unwanted immune responses in gene therapy.

14 y to confirm the safety and efficacy of this gene therapy.

15 in novel therapeutic interventions including gene therapy.

16 ideration for the future development of XLRS gene therapy.

17 safety and efficacy of a novel intracerebral gene therapy.

18 levant for a broader clinical application of gene therapy.

19 eatly improved their properties for systemic gene therapy.

20 hematopoietic stem cell transplantation and gene therapy.

21 ruses (AAVs) are promising vectors for human gene therapy.

22 anding of the biological barriers to inhaled gene therapy.

23 NA to target cells is critical to successful gene therapy.

24 ous gene and HLA haplotype on the outcome of gene therapy.

25 ly engineering minimized dystrophins for DMD gene therapy.

26 ing is essential for biomedical research and gene therapy.

27 lease of therapeutic biomolecules in ex vivo gene therapy.

28 been long considered a logical candidate for gene therapy.

29 -Aldrich syndrome patients treated with HSPC gene therapy.

30 of concept for point-of-care delivery of HSC gene therapy.

31 lability and standardized manufacture of HSC gene therapy.

32 nsider when selecting candidates for retinal gene therapy.

33 ce the therapeutic activity of an anticancer gene therapy.

34 elopment of a clinical rAAV candidate for CF gene therapy.

35 were analyzed as therapeutic targets for TCR gene therapy.

36 revealing the potential importance of UTR in gene therapy.

37 ted refractive errors may be amenable to AAV gene therapy.

38 ports the use of foamy virus as a vector for gene therapy.

39 assess thoroughly the safety of BNP116.I-1c gene therapy.

40 (rd6) mice via adeno-associated viral (AAV) gene therapy.

41 e versatile tools in functional genomics and gene therapy.

42 demonstrated outstanding potential for human gene therapy.

43 opment of ALV-based vectors for use in human gene therapy.

44 ome makes them a crucial element of clinical gene therapy.

45 n designing safe and efficacious vectors for gene therapy.

46 of microbubble-enhanced sonoporation-induced gene therapies.

47 new avenues for basic research and targeted gene therapies.

48 as a bioengineering tool for anticancer and gene therapies.

49 ledge, AAV2-hRPE65v2 is the first successful gene therapy administered to the contralateral eye.

50 indicate the efficacy of a new combinatorial gene therapy aimed at rescuing DA function and related p

51 f obstructive lung diseases has made inhaled gene therapy an attractive alternative to the current st

52 ing challenges toward the development of HSC gene therapies and discuss lessons they provide for the

53 gels may provide a versatile tool to combine gene therapy and biomaterials for applications in regene

54 ng will be useful to improve vaccine design, gene therapy and cancer treatment.

55 we summarize the available published data on gene therapy and discuss the challenges, opportunities,

56 erapeutics, specifically for applications in gene therapy and DNA vaccination.

57 peutic options are in development, including gene therapy and gene editing.

58 ent, and new delivery modalities in cell and gene therapy and oligonucleotide approaches are yielding

59 saic mutations is particularly important for gene therapy and precision genome editing.

60 ctor in leukemogenic potential of retroviral gene therapy and underscore the importance of cytoreduct

61 ntially impacting responses to DNA vaccines, gene therapy, and autoimmune disease pathogenesis.

62 baseline and at 3, 6, 9, and 12 months after gene therapy, and every 6 months thereafter for one furt

63 to revolutionize diagnostics, drug delivery, gene therapy, and many other areas of research.

64 cerebral haemorrhage) at days 3-7 after AAV2 gene therapy, and we assessed adverse events during the

65 d this delivery route hold great promise for gene therapy applications in both cochlear and vestibula

66 utility of integrating retroviral vectors in gene therapy applications.

67 g its activity to generate a potent tool for gene therapy applications.

68 y neurons of the dorsal root ganglia using a gene therapy approach and found an enhancement or reduct

69 esults pave the way toward a novel non-viral gene therapy approach for DMD using PB transposons under

70 In summary, we demonstrate an HSC-based gene therapy approach for IFNgammaR1 deficiency, which p

71 endogenous FOXP3 regulation for an HSC-based gene therapy approach for IPEX syndrome.

72 overexpression of IL-10 in microglia using a gene therapy approach significantly delayed disease onse

73 To develop a gene therapy approach targeting myelinating Schwann cell

74 we developed a hematopoietic stem cell (HSC) gene therapy approach using lentiviral vectors that expr

75 hese dogs, we determine the feasibility of a gene therapy approach using liver-directed, adeno-associ

76 Using a gene therapy approach, here we show that increasing brai

77 urther clinical development of this nonviral gene therapy approach.

78 f cytoreductive conditioning in this type of gene therapy approach.

79 sing strategy for the development of a novel gene therapy approach.

80 easing expression of CRRY in the RPE using a gene therapy approach.

81 developing long-lasting, safe, and versatile gene therapy approaches based on engineering epidermal p

82 enes and mutations that cause RP, corrective gene therapy approaches currently in development may pro

83 Various forebrain-directed gene therapy approaches have only had limited success in

84 ated that targeting oncogene expression with gene therapy approaches is feasible in patients.

85 ments for hearing loss, we sought to advance gene therapy approaches to treat genetic deafness.

86 targets has spurred clinical development of gene therapy approaches to treat patients with malignant

87 Although conventional gene therapy approaches typically involve the addition o

88 ected cells.IMPORTANCE The majority of human gene therapy approaches utilize HIV-1- or murine leukemi

89 cal studies, several clinical trials for SCD gene therapies are now open.

90 oteostasis network using small molecules and gene therapy are under development, and promise interest

91 emonstrate the therapeutic potential of PUMA gene therapy as a local treatment in various forms of ar

92 eristics to assess the potential for retinal gene therapy as a means of preventing severe visual loss

93 These data emphasized Ang-(1-9) gene therapy as a potential new strategy in the context

94 These findings demonstrate that gene therapy-based interventions targeting the beta2-AR

95 ently the leading candidates for virus-based gene therapies because of their broad tissue tropism, no

96 Cs appear to be an ideal target for platelet gene therapy because they can differentiate into megakar

97 might allow additional PIDs to be treated by gene therapy because they will allow the endogenous gene

98 n biomedical fields including drug delivery, gene therapy, biosensors, and tissue engineering applica

99 eno-associated virus (AAV) holds promise for gene therapy but faces critical barriers on account of i

100 Viral vectors are effective tools in gene therapy, but their limited packaging capacity can b

101 l combinatorial adeno-associated viral (AAV) gene therapy by expressing DAT selectively in DA neurons

102 However, many challenges remain before gene therapy can advance to a clinically relevant AF tre

103 Therefore, mutation-adapted U1 snRNA gene therapy can be a promising method to treat genetic

104 ardiac contractility that when combined with gene therapy can be employed as a therapeutic strategy f

105 Initial lung gene therapy clinical trials occurred in the early 1990s

106 allenge that restricts patient enrollment in gene therapy clinical trials using recombinant AAV vecto

107 More than 1800 gene therapy clinical trials worldwide have targeted a w

108 AAV) have been utilized in a large number of gene therapy clinical trials, which have demonstrated th

109 of OPMD with an adeno-associated virus-based gene therapy combining complete knockdown of endogenous

110 As the development of this promising gene therapy continues, safety considerations are a high

111 randomly assigned (3:1) to treatment and non-gene therapy control groups.

112 ever, the efficiency, safety, and cost of LV gene therapy could be ameliorated by enhancing target ce

113 s MPM tumor growth and evaluate whether EPCR gene therapy could suppress the progression of MPM in a

114 precursor nicotinamide (vitamin B3), and/or gene therapy (driving expression of Nmnat1, a key NAD(+)

115 Tissue engineering, gene therapy, drug screening, and emerging regenerative

116 science and translational medicine including gene therapy, due to the versatility in its cell and org

117 Transient post-gene therapy dyskinesia occurred in all patients but was

118 strate that hematopoietic stem cell-mediated gene therapy effectively terminates antigen-specific mem

119 t adeno-associated viral vector (rAAV)-based gene therapy encouraged us to reexplore an rAAV approach

120 pport the use of foamy virus as a vector for gene therapy, especially when strong enhancers/promoters

121 ascularization, adeno-associated virus (AAV) gene therapy, exploiting a natural immune tolerance mech

122 f clinical efficacy is still well behind the gene therapy field, multiple programs investigating rege

123 uct remain an active area of concern for the gene therapy field.

124 cell specificity, are major barriers in the gene therapy field.

125 This work enables the design of safer TCR gene therapies for cancer immunotherapy.

126 complex auditory function, may enable future gene therapies for hearing and balance disorders.

127 Efforts to develop gene therapies for hearing loss have been hampered by th

128 ld contribute to the development of improved gene therapies for various neurological disorders by exp

129 monstrate clinical therapeutic efficacy from gene therapy for ADA-deficient SCID, with an excellent c

130 elis is a European Medicines Agency approved gene therapy for ADA-SCID patients without a suitable bo

131 t adeno-associated virus (AAV)-mediated RNAi gene therapy for ALS.

132 evaluated the preclinical potential of rAAV gene therapy for CF to restore chloride and fluid secret

133 b Escort Protein 1 is described as a part of gene therapy for choroideremia.

134 Nevertheless, before the application of gene therapy for coagulation disorders becomes widesprea

135 Gene therapy for cystic fibrosis using non-viral, plasmi

136 Gene therapy for dry mouth disorders has transitioned in

137 Gene therapy for genetic deafness is a promising approac

138 to 71 years underwent unilateral subretinal gene therapy for genetically confirmed choroidermeia.

139 therapy and should be further explored as a gene therapy for GM2 gangliosidoses.

140 Gene therapy for hemophilia B aims to ameliorate bleedin

141 lenged this approach as a possible HSC-based gene therapy for IPEX.

142 Allotopic gene therapy for LHON at low and medium doses seems to b

143 To test the potential efficacy of gene therapy for NPC1, we constructed adeno-associated v

144 uences that may contribute to the success of gene therapy for ocular disorders, the role of versican,

145 This clinical trial extends the reach of TCR gene therapy for patients with metastatic cancer.

146 Gene therapy for patients with this disorder is complica

147 hematopoietic stem cell transplantation and gene therapy for primary immunodeficiency have had relat

148 ing may usher in a second-generation form of gene therapy for the beta-globin disorders.

149 view summarizes the progress of AAV-mediated gene therapy for the hemophilias, along with its upcomin

150 tent Stem Cells (iPSCs) provides a potential gene therapy for this debilitating disease.

151 The first attempts at gene therapy for WAS using a Upsilon-retroviral vector i

152 Lentiviral vector HSPC gene therapy generates a human hematopoietic system stab

153 Gene therapy (GT) has offered immense hope to individual

154 We investigated the medium-term outcome of gene therapy (GT) in 18 patients with ADA-SCID for whom

155 antation is the standard treatment; however, gene therapy (GT) might represent a valid alternative, e

156 Viral vector mediated gene therapy has become commonplace in clinical trials f

157 Gene therapy has been developed as a method to perform a

158 Gene therapy has been pursued as a promising strategy to

159 Haematopoietic stem cell (HSC) gene therapy has demonstrated potential to treat many di

160 Retroviral gene therapy has proved efficacious for multiple genetic

161 iated viral (AAV) vectors for liver-directed gene therapy has shown considerable success, particularl

162 Gene therapy has the potential for a definitive cure, an

163 Gene therapy has the potential to provide innovative tre

164 h any drug development strategy, delivery of gene therapy has to be consistent and predictable in eac

165 espite over two decades of intensive effort, gene therapy has yet to help patients with CF or any oth

166 Recent clinical trials of gene therapy have implicated the need of an alternative

167 Gene therapy holds promise for the treatment of many pat

168 rs have made great progress in their use for gene therapy; however, fundamental aspects of AAV's caps

169 The endothelium-targeted gene therapy improved the integrity of the BBB.

170 should be good targets for future localized gene therapies in patients.

171 ess of adeno-associated virus (AAV)-mediated gene therapy in clinical trials is promising, challenges

172 ards clinical trials of rAAV-microdystrophin gene therapy in DMD patients.

173 We examined the efficacy of G6PT gene therapy in G6pt-/- mice using recombinant adeno-ass

174 utic benefit of a brain endothelial-targeted gene therapy in IP.

175 The favorable response to gene therapy in Mfrp (rd6) /Mfrp (rd6) mice suggests hyp

176 The application of gene therapy in osteoarthritis offers insights because i

177 region has precluded improved outcomes with gene therapy in patients with hemophilia A.

178 e, or off-target organ damage by BNP116.I-1c gene therapy in pigs.

179 s and their vectors have shown potential for gene therapy in preclinical studies.

180 for the successful application of non-viral gene therapy in skin disease.

181 romising new delivery platform for localized gene therapy in the brain.

182 Importantly, CRTC1 gene therapy in the hippocampus ameliorates context memo

183 Major advances in gene therapy include the emergence of recombinant adeno-

184 mponents for successful execution of inhaled gene therapy, including gene delivery systems, primary p

185 Such an approach would also be useful for gene therapy, including the treatment of neurodegenerati

186 w the most important aspects of using SB for gene therapy, including vectorization as well as genomic

187 me intron as retroviral integration sites in gene therapy-induced T-ALL, suggesting that such events

188 ent increase in biopharmaceutical funding in gene therapy, industry partners are requiring their acad

189 s in biotechnology, biocomputing, and modern gene therapy interventions are often based on plasmids o

190 or hematopoietic stem/progenitor cell (HSPC) gene therapy, involving the transplantation of ex vivo g

191 Organic nanoparticle-based (ONP) gene therapy is a potential strategy to cure human cance

192 Somatic gene therapy is a promising approach for treating otherw

193 In situ gene therapy is a promising approach to address the limi

194 Gene therapy is a promising treatment strategy.

195 Adeno-associated virus (AAV)-mediated gene therapy is currently being pursued as a treatment f

196 sy, less invasive injection route for ocular gene therapy is met by intravitreal delivery, but delive

197 ng iatrogenic macular detachment for retinal gene therapy is not well characterized in those with rel

198 Gene therapy is providing exciting new treatment options

199 The use of viral vectors for inner ear gene therapy is receiving increased attention for treatm

200 effective modulation of immune responses in gene therapy is still long; the determinants of the bala

201 question in DMD pathogenesis and dystrophin gene therapy is whether muscle health depends on continu

202 e optimal intervention window for subretinal gene therapy is within the first 2 to 3 decades of life.

203 The main hurdle to using lipoplexes in gene therapy lies in their immunostimulatory properties,

204 our study suggests that even low-efficiency gene therapy may achieve stable survival of rescued phot

205 y results of this study suggest that Lenti-D gene therapy may be a safe and effective alternative to

206 rcuits, supporting the concept that neonatal gene therapy may prevent the functional abnormalities th

207 results provide the first demonstration that gene therapy may represent a therapeutic option for NPC1

208 Transplantation and gene therapy may serve to replace or resurrect dead or i

209 Both transgenic and AAV2/8 gene therapy-mediated ablation of Sirt6 in rods provided

210 Notably, gene therapy-mediated PINK1 overexpression promotes the

211 Replacing Nemo through gene therapy might provide therapeutic benefits.

212 cell receptor and chimeric antigen receptor gene therapy models.

213 Our findings have clear implications for gene therapy of airway disorders where plasmid DNA trans

214 sults open up new perspectives for efficient gene therapy of cochlear and vestibular disorders by sho

215 e corrected HSPCs, opening new prospects for gene therapy of FA patients.

216 ombinant HBoV1 vectors, a promising tool for gene therapy of lung diseases.

217 Hematopoietic stem cell transplantation or gene therapy offer a cure, but despite successful replac

218 ids are generally regarded as the payload in gene therapy, often requiring a carrier for intracellula

219 The authors evaluated effects of Ang-(1-9) gene therapy on myocardial structural and functional rem

220 y benefit from IKACh suppression achieved by gene therapy or selective pharmacological inhibition.

221 ntricular routes or with fusion proteins, or gene therapy) or applicable to more than one lysosomal d

222 rs are already used for liver-directed human gene therapy, our strategy has potential for clinical tr

223 n that should inform methods for efficacious gene therapy over a broad range of applications.

224 fellow eye improvement in our acute group 2 gene therapy patients of 0.96 was more than that observe

225 e primary efficacy endpoint: 12 months after gene therapy, PDMS-2 scores were increased by a median o

226 or BTICs and establishes a flexible nonviral gene therapy platform with the capacity to channel multi

227 microenvironment and to develop the cell and gene therapy potential of KC transplantation.

228 Our novel findings showed that Ang-(1-9) gene therapy preserved left ventricular systolic functio

229 program a frontrunner for the first approved gene therapy product in the United States.

230 e of several programs presents challenges to gene therapy product manufacturing.

231 Similar to gene therapy, progress in regenerative or stem cell-base

232 These findings indicate that telomerase gene therapy represents a novel therapeutic strategy to

233 lly increasing CRP expression using targeted gene therapy represents a potential treatment strategy f

234 development of recombinant viral vectors for gene therapy require that products are well characterize

235 The widespread clinical implementation of gene therapy requires the ability to stably integrate ge

236 Outside of the clinic, gene therapy research is evolving to overcome the poor r

237 The slow progress in gene therapy research, high incidence of technical compl

238 These analyses showed gene therapy restored retinal function and normalized ax

239 Surprisingly, forebrain-directed gene therapy resulted in essentially no PPT1 activity in

240 lthough there are promising results in human gene therapy, RP is a genetically diverse disorder, such

241 Here, we show that GALGT2 gene therapy significantly reduces muscle pathology in F

242 ults suggest that clinical approaches for FA gene therapy similar to those used in this study will fa

243 ted of a subretinal injection of 0.1 mL of a gene therapy solution containing 1 x 1011 viral particle

244 Current DMD gene therapy strategies rely on the expression of intern

245 olyamine, spermine, in affected animals, and gene therapy studies demonstrated that reduction of CSF

246 Previous Fanconi anemia (FA) gene therapy studies have failed to demonstrate engraftm

247 we have started to see examples of clinical gene therapy successes.

248 Rescue of the STGD1 phenotype by AAV-CRRY gene therapy suggests that complement attack on the RPE

249 tumor cell apoptosis, and intrapleural EPCR gene therapy suppresses MPM progression.

250 entered around the development of autologous gene therapies targeting private somatic mutations.

251 d definitive genomic diagnoses and potential gene therapy targets.

252 This treatment is the first gene therapy that reverses disability after stroke when

253 Given the many target diseases for gene therapy, there is enormous potential for this appro

254 extended half-life to nonfactor products and gene therapy, these innovative approaches have the poten

255 estigate the clinical benefits and safety of gene therapy through infusion of adeno-associated virus

256 ractical applications of BOEC: their use for gene therapy, tissue engineering, assessment of mutant g

257 cy diseases and encourage the development of gene therapies to counter such diseases.

258 safe and non-invasive strategy for targeted gene therapy to the brain.

259 d stimulate further research into the use of gene therapy to treat patients with heart failure and he

260 (AAV1), the first viral vector approved as a gene therapy treatment, and its closely related AAV6, si

261 -based vector has been approved as the first gene therapy treatment.

262 poietic system, but roughly half of clinical gene therapy trial protocols using gammaretroviral vecto

263 Success of ongoing clinical gene therapy trials depends on many factors such as sele

264 t an overview of recent progress in clinical gene therapy trials of the MD's and touch upon promising

265 of the most commonly used viral vectors for gene therapy trials, and demonstrate their potential use

266 n combination with results of other ADA-SCID gene therapy trials, suggest that disease background may

267 failure and help inform the design of future gene therapy trials.

268 Autologous gene therapy using a lentiviral vector is a viable strat

269 Furthermore, a gene therapy using a nanoparticle formulated with an siR

270 cytes and thus the outcome of liver-directed gene therapy using AAV vectors and showed in a proof-of-

271 Hepatic arginase 1 gene therapy using adeno-associated virus rescued nearly

272 Gene therapy using autologous HSCs should avoid these li

273 uces HbF expression, providing the basis for gene therapy using gene editing tools.

274 Gene therapy using highly functional microdystrophin gen

275 Gene therapy using nonintegrating viruses such as adeno-

276 Intravitreal injection with the gene therapy vector AAV2(Y444,500,730F)-P1ND4v2 into 1 e

277 an adeno-associated virus serotype 9 (AAV9) gene therapy vector or recombinant protein, resulted in

278 ent parvovirus that is extensively used as a gene therapy vector.

279 apeutic transgene expression from retroviral gene therapy vectors by epigenetic defence mechanisms re

280 ation by using adeno-associated virus (AAV)9 gene therapy vectors carrying the telomerase Tert gene i

281 eno-associated viruses (AAVs) are attractive gene therapy vectors due to their low toxicity, high sta

282 Generating disease responsive gene therapy vectors requires knowledge of the activatio

283 Efficient production of AAV-based gene therapy vectors requires optimal Rep expression lev

284 g developed as oncolytic therapeutics and as gene therapy vectors.

285 These data suggest that melanopsin gene therapy via a subretinal route may be a viable and

286 or vascular endothelial growth factor (VEGF) gene therapy via adeno-associated viral type-2 (AAV2) ve

287 al trial findings support the feasibility of gene therapy via recombinant adeno-associated viral vect

288 ses, and may also modulate the efficiency of gene therapy viral vectors.

289 d drug enhancement of adeno-associated virus gene therapy, which could result in safe and effective t

290 be used for permanent full-length dystrophin gene therapy, which presents a significant advancement i

291 ease the safety and benefit of megakaryocyte gene therapy will be discussed.

292 Widespread application of gene therapy will depend on the development of simple me

293 Gene therapy with a virus encoding Salv short hairpin RN

294 Immune responses in gene therapy with adeno-associated virus (AAV) vectors h

295 ts to secrete WNT16B, enabling potent cancer gene therapy with few side effects.

296 Gene therapy with genetically modified human CD34(+) hem

297 Its gene therapy with the TRAIL suicide gene effectively ind

298 We have demonstrated gene therapy with this vector by restoring dystrophin ex

299 reatment was also beneficial to AAV-mediated gene therapy with transfer of micro-dystrophin cDNA into

300 gly, clinical trials have shown that inhaled gene therapy with various viral vectors and non-viral ge