ライフサイエンスコーパス: gene therapy

1 plication of genetic engineering technology (gene therapy).

2 as a pathogen and emerging vector for human gene therapy.

3 ariants is required for disease modeling and gene therapy.

4 adenovirus-mediated angiopoietin-1 (Angpt1) gene therapy.

5 r instead led to safer but still efficacious gene therapy.

6 minate anti-AAV antibodies in the context of gene therapy.

7 at could provide potential targets for novel gene therapy.

8 they represent a major limitation to in vivo gene therapy.

9 improve the use of this vector as a tool for gene therapy.

10 st reliable gene delivery vehicles for human gene therapy.

11 he way for treatment with emerging inner ear gene therapy.

12 rs may further facilitate the success of AAV gene therapy.

13 vercome pre-existing antibodies to AAV-based gene therapy.

14 ssociated virus (AAV) hold great promise for gene therapy.

15 n 20% of liver HGD to correct the disease in gene therapy.

16 oliferation or leukaemia were reported after gene therapy.

17 ting from F8 mutations, can only be cured by gene therapy.

18 ers involving motor function, improved after gene therapy.

19 pecies, both for fundamental science and for gene therapy.

20 nature, HB is one of the primary targets for gene therapy.

21 g candidate for rAAV-mediated liver-targeted gene therapy.

22 g the efficacy and safety of muscle-directed gene therapy.

23 utic developments, including drug design and gene therapy.

24 and the potential applications for inner ear gene therapy.

25 pivotal technique in biomedical research and gene therapy.

26 designing new recombinant viral vectors for gene therapy.

27 s, limitations, and progress in clinical AAV gene therapy.

28 studies will confirm the safety of LV-based gene therapy.

29 us (AAV) is a leading vector for virus-based gene therapy.

30 TI) was performed before and 12 months after gene therapy.

31 s for the development of new AAV vectors for gene therapy.

32 s for vaccination, cancer immunotherapy, and gene therapy.

33 nology and has a great potential for in vivo gene therapy.

34 drome who received lentiviral vector-derived gene therapy.

35 le to allogeneic HSC transplantation and HSC gene therapy.

36 of vehicles that are clinically relevant for gene therapy.

37 kilogram or 6x10(13) vg per kilogram of the gene therapy.

38 r delivery is required, such as oncology and gene therapy.

39 IDX1 and ADA deficiency as a kind of natural gene therapy.

40 rs and other diseases such as cancer through gene therapy.

41 ates for RMCE reactions for future potential gene therapy.

42 generation of cell type-specific drivers for gene therapy.

43 lls is a prevalent challenge in the field of gene therapy.

44 standing regulatory processes and developing gene therapies.

45 assess the need for and promise of specific gene therapies.

46 vectors are preeminent in emerging clinical gene therapies.

47 , for production of recombinant proteins and gene therapies.

48 p safer and more efficient viral vectors for gene therapies.

49 adeno-associated virus (AAV) vector-mediated gene therapies.

50 molecule and biologic therapies, devices and gene therapies.

51 education on the various aspects of cell and gene therapies.

52 d a major milestone in the field of cell and gene therapies.

53 (1) Knowledge and understanding of cell and gene therapies, (2) Acceptance of cell and gene therapie

54 d gene therapies, (2) Acceptance of cell and gene therapies (3) Understanding of risk and benefits of

55 es its contractibility and explain why SERCA gene therapy, a change in calcium handling to treat hear

56 ventions targeting motor symptoms, including gene therapy, adaptive deep brain stimulation (DBS) and

57 As gene therapy advances, enrollment into the International

58 nti-tissue factor pathway inhibitor, and the gene therapy aimed at improving the patient's quality of

59 ChABC gene therapy alone did not enhance motoneuron survival,

60 tches may improve the safety and efficacy of gene therapies and broaden their use.

61 , we review the most recent advances in HSPC gene therapy and discuss emerging strategies for using H

62 re treated with adeno-associated viral (AAV) gene therapy and followed for up to 10 years.

63 diseases, and might be especially suited to gene therapy and gene editing settings in which preserva

64 Moreover, gene therapy and gene editing technologies are being use

65 es are already established in the clinic for gene therapy and immunotherapy, and inactivated viruses

66 he host immune response against AAV-mediated gene therapy and influence the course of septicaemia.

67 -associated viral and lentiviral vectors for gene therapy and lipid nanoparticle and other non-viral

68 dysregulated in oral cancer patients, using gene therapy and repurposing an available drug to effect

69 show great promise and, in combination with gene therapy and surgical techniques, have the potential

70 With the advent of gene therapy and the numerous ongoing clinical trials fo

71 e of SAdVs as gene delivery vectors in human gene therapy and vaccines, selected to avoid preexisting

72 ot mean alone) is crucial for the success of gene therapies, and that long-term rather than short int

73 c modalities (combination therapies, ex-vivo gene therapy, and in-vivo gene therapy) for a target pro

74 e limited attempts to treat CF using in vivo gene therapy, and low correction levels have hindered ex

75 exploration of strategies in electrotherapy, gene therapy, and optogenetics.

76 The future of gene therapy appears promising for the GSDs, promising t

77 proaches to engineer recombinant capsids for gene therapy applications have focused on rational desig

78 ttention should be given to liver changes in gene therapy applications when genes affecting cholester

79 enoviruses have many attractive features for gene therapy applications.

80 the possibility of using such synergism for gene therapy applications.

81 overy of capsids for use in neuroscience and gene-therapy applications.

82 results further support the application of a gene therapy approach as a novel treatment for OPMD in h

83 the expression of these three microRNAs in a gene therapy approach displays significant anticancer sy

84 One Sentence Summary: A gene therapy approach for Alzheimer's disease using aden

85 This HSPC gene therapy approach has potential for clinical transla

86 Using a highly novel gene therapy approach in a canine, rapid atrial pacing m

87 h-risk germ-line mutations, the in vivo HSPC gene therapy approach is a promising strategy that addre

88 An innovative intrathymic gene therapy approach that directly targets the thymus m

89 cal approval pipeline, there is not a single gene therapy approach that has worked for the heart.

90 We have developed a simple in vivo HSPC gene therapy approach that involves HSPC mobilization an

91 l advance in the development of our melanoma gene therapy approach.

92 Gene therapy approaches are being deployed to treat rece

93 Anti-VEGF gene therapy approaches can create significantly differe

94 c knockdown and provide proof of concept for gene therapy approaches for dominant neuromuscular disea

95 hair cell loss, and may help identify future gene therapy approaches for restoring hearing.

96 Although clinical gene therapy approaches using lentiviral vectors have pr

97 liosidosis potentially more safely than with gene therapy approaches.

98 gets in the lung and testing the efficacy of gene therapy approaches.

99 f HB has been shown in clinical trials using gene therapy approaches.

100 nt insight for improving transplantation and gene therapy approaches.

101 is for assessing the price ceiling of myriad gene therapy approaches.

102 ted both small-molecule readthrough drug and gene-therapy approaches for this "disease-in-a-dish" app

103 and that long-term rather than short intense gene therapies are more likely to beneficially impact mt

104 ances in cell-based, protein replacement and gene therapies are paving the way for clinical successes

105 public opinions and experiences of cell and gene therapy are required.

106 Hematopoietic stem cell transplantation and gene therapy are the only curative treatments available,

107 these results provided further indication of gene therapy as a possible effective treatment option fo

108 ochondrial disease, both small molecules and gene therapies, as well as methods to prevent transmissi

109 In this study, we developed gene therapies based on 3 longevity associated genes (fi

110 e family counseling and future enrollment in gene therapy-based treatments.

111 However, recent advances in gene therapy, better outcomes with stem cell transplanta

112 ciated virus (AAV) is a promising vector for gene therapy, but its broad tropism can be detrimental i

113 Gene therapy by expression constructs or down-regulation

114 of human alpha-mannosidosis by intravascular gene therapy', by Yoon et al. (doi:10.1093/brain/awaa161

115 Adeno-associated virus (AAV)-based gene therapies can restore endogenous factor VIII (FVIII

116 rapy, the longest post-mortem trophic factor gene therapy cases reported to date.

117 While clinical gene therapy celebrates its first successes, with severa

118 ast experience gained in the several cardiac gene therapy clinical trials that had the goal of induci

119 nd have implication for ongoing hemophilia A gene-therapy clinical trials.

120 g computational drug repositioning, cell and gene-therapy, clustered regularly interspaced short pali

121 herapy following stromal deactivation by RLN gene therapy completely cured established CRC liver meta

122 Complementary AAV-based gene therapies constitute rationally-designed strategies

123 The use of non-viral vectors for in vivo gene therapy could drastically increase safety, whilst r

124 indicate that therapeutic effects of retinal gene therapy decrease gradually as treatments are given

125 e of engineered bacteriophages-akin to human gene therapies delivered by viral vectors.

126 ot avulsion injuries, we combined timed GDNF gene therapy delivered to the proximal nerve roots with

127 A number of intraparenchymally delivered gene therapies designed to modify underlying disease and

128 ed human aromatic L-amino acid decarboxylase gene therapy development programme.

129 We also emphasize a few areas of cardiac gene therapy development that hold great promise for the

130 aged 1.67-8.42 years) enrolled in the study, gene therapy did not affect macroscopic structure.

131 Gene therapy displacement of endogenous MCUb with a domi

132 The proposed design is a prototype for a gene therapy DNA machine that cleaves a housekeeping gen

133 triking clinical proof of concept, the first gene therapy drugs coming onto the market, and the emerg

134 AAV capsid to gain desirable properties for gene therapy (e.g., tropism, reduced immunogenicity, and

135 cle (NP) technologies to further improve the gene therapy efficacy by prolonging the release of nucle

136 Gene therapy efforts have focused on treating the lung,

137 elivery system, limiting most of the current gene therapy efforts to ex vivo editing of extracted cel

138 low correction levels have hindered ex vivo gene therapy efforts.

139 antation of lumbar ventral roots, timed GDNF-gene therapy enhanced motoneuron survival up to 45 weeks

140 et tissues, which has guided the planning of gene therapy experiments.

141 ntributed substantially to the growth of the gene therapy field.

142 Despite the success of recent RPE65 gene therapy, follow-up studies show that patients conti

143 ic stem and progenitor cell-based lentiviral gene therapy following myeloablative conditioning in fir

144 equential administration of FOLFOX and IL-12 gene therapy following stromal deactivation by RLN gene

145 have renewed the field's focus on developing gene therapies for the 10% of CF patients these modulato

146 nform efforts to develop pharmacological and gene therapies for treating cocaine use disorders.

147 d discuss emerging strategies for using HSPC gene therapy for a range of diseases.

148 potential of these reversible on-switches in gene therapy for anemia of chronic kidney disease(6), we

149 e focus of this review is the development of gene therapy for glycogen storage diseases (GSDs).

150 dings bring us closer to a widely applicable gene therapy for hemoglobin disorders.

151 e clinical development of liver-directed AAV gene therapy for hemophilia A, while emphasizing the imp

152 foundation for the first clinical trials of gene therapy for junctional and dystrophic epidermolysis

153 Gene therapy for Leber congenital amaurosis (LCA) is bec

154 e biomedical field are in the development of gene therapy for LSDs.

155 which increases the need for development of gene therapy for NPC1.

156 ults identify a bone-targeting rAAV-mediated gene therapy for osteoporosis.

157 s will likely inform productive paths toward gene therapy for other complex genetic disorders, while

158 One-by-one, gene therapy for primary immune deficiencies is being br

159 Nevertheless, the first attempts of gene therapy for SCID X1 were associated with insertiona

160 ed nanoparticles are promising for non-viral gene therapy for Stargardt disease and can be expended f

161 Gene therapy for this disease presents inherent hurdles

162 These results emphasize the promise of gene therapy for treating diverse age-related ailments a

163 ing clonal tracking in patients treated with gene therapy for Wiskott-Aldrich syndrome (WAS) and beta

164 ler cells) in patients having undergone HSPC gene therapy for Wiskott-Aldrich syndrome or beta hemogl

165 therapies, ex-vivo gene therapy, and in-vivo gene therapy) for a target product profile for an HIV cu

166 ped and are commonly referred to as germline gene therapy (GGT).

167 Gene therapy (GT) has tremendous potential for the treat

168 Over 3 decades, gene therapy has advanced from a logical idea to becomin

169 Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to cor

170 ematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective treatment modal

171 Retinal gene therapy has had unprecedented success in generating

172 oration of dopamine levels in the putamen by gene therapy has led to significant improvement in motor

173 Retinal gene therapy has shown great promise in treating retinit

174 that are currently in clinical development, gene therapy holds the promise of a lasting cure with a

175 hat can potentially be modulated directly by gene therapy, if we can achieve RGC specific gene target

176 our emerging oncology-associated fields: (i) gene therapy, (ii) immunotherapy, (iii) extracellular ve

177 ign and library-based evolution for clinical gene therapy.IMPORTANCE Clinical gene therapy with recom

178 the way for broader clinical applications of gene therapies in dermatology.

179 tages and examined the long-term efficacy of gene therapy in a mouse model of ciliopathy.

180 Subretinal gene therapy in Abca4(-/-) mice using ECO/pRHO-ABCA4 and

181 esence of limited time window for successful gene therapy in certain retinal degenerations.

182 le can be used to monitor the effect of CLN2 gene therapy in future trials.

183 y evidence of the benefits of FLRL2-mediated gene therapy in NAFLD.

184 These results validate the feasibility of gene therapy in preventing and restoring metabolic homeo

185 is a growing need for monitoring or imaging gene therapy in the central nervous system (CNS).

186 Gene therapy increased the life span of treated animals,

187 Gene therapy inhibition of RAR increases innate and lear

188 mbination treatment of macitentan and ET(B)R gene therapy inhibits invasion, but not proliferation, i

189 ligonucleotides, deep brain stimulation, and gene therapy into the clinic within the next decade or s

190 Viral gene therapy is a means of delivering genes to replace m

191 ted haemopoietic stem/progenitor cell (HSPC) gene therapy is a potentially curative treatment that re

192 months follow-up, suggesting that autologous gene therapy is a promising approach for CGD patients.

193 These data demonstrate that brain-directed gene therapy is a valid strategy to treat the neurodegen

194 diseases linked to ciliary dysfunction, and gene therapy is an attractive treatment option to preven

195 Macrophage-targeted gene therapy is an effective strategy for regulating mac

196 Gene therapy is at an inflection point.

197 Hematopoietic stem cell (HSC) gene therapy is being evaluated for hemoglobin disorders

198 These therapies show that gene therapy is both safe and effective, with the potent

199 ly, we provide in vitro evidence that mChABC gene therapy is equally or more effective at producing t

200 Gene therapy is now being trialled as a therapeutic opti

201 Gene therapy is one of the most promising medical fields

202 Adeno-associated viral vector (AAV)-based gene therapy is thus emerging as a potential treatment f

203 econstitution during inflammation, remain if gene therapy is to be extended to more complex diseases

204 While viral based gene therapy is under development, it is important to in

205 Adeno-associated virus (AAV)-mediated gene therapy is under investigation as a therapeutic opt

206 Although the efficacy of retinal gene therapy is well established by multiple proof-of-co

207 Mutations, genetic manipulation, and gene therapies may produce cells where different types o

208 plies that the long-term efficacy of retinal gene therapy may depend on not only the timing of treatm

209 nly considered for rare inherited disorders, gene therapy may open treatment opportunities for more c

210 at least 6 months after receiving BCH-BB694 gene therapy; median follow-up was 18 months (range, 7 t

211 This study provides the first evidence that gene therapy mediated by AAV vectors can be used for tre

212 The first gene therapy medicines have been licensed for marketing

213 ologicals in oral immunotherapy, advances in gene therapy, multifood therapy, novel diagnostics in di

214 Gene therapy needs to replace deficient enzymes in targe

215 d to develop the next-generation vectors for gene therapy of muscle disorders, given the relatively m

216 sent work develops a plasmid DNA approach to gene therapy of NPC1 using Trojan horse liposomes (THLs)

217 HDAd5/35++ vectors for in vivo gene therapy of thalassemia had a unique capsid that tar

218 Cell and gene therapies offer opportunities for treating disease

219 Currently, gene therapy offers a novel approach for treating monoge

220 peutic applications in the clinical setting, gene therapy offers several advantages over traditional

221 an be used to select among a growing list of gene therapy options to maximize safety and efficacy whi

222 Intramuscular gene therapy post-disease onset using an adeno-associate

223 nd telomerase-deficient mice with telomerase gene therapy prevented the onset of lung profibrotic pat

224 Current thalassemia gene therapy protocols require the collection of hematop

225 Recent developments in immunotherapies and gene therapies provide renewed hope in advancing efforts

226 t of whether experimental therapies, such as gene therapy, provide a functional benefit.

227 Ongoing advances in gene therapy, regenerative therapy and cell augmentation

228 We previously demonstrated that gene therapy represents a feasible therapeutic tool for

229 Collectively, the targeted RLN gene therapy represents a highly efficient, safe, and ve

230 vitro and in WAS patients following clinical gene therapy restores autophagic flux and is dependent o

231 In situ gene therapy resulted in complete inhibition of tumor pr

232 Gene therapy results in an improvement in median surviva

233 liver metastasis models, we confirm the RLN gene therapy results in significant inhibition of metast

234 DTI before gene therapy revealed lower total mean fractional anisot

235 n-conditioned patients with FA supports that gene therapy should constitute an innovative low-toxicit

236 sting intervention with retina-specific CLN2 gene therapy should occur ideally before or as early as

237 A literature survey also indicates that gene therapy, stem cell therapy, and target discovery th

238 In this review, we summarize these gene therapy strategies and ongoing clinical trials.

239 As gene therapy strategies strive toward dystrophin restora

240 Stabilizer cell gene therapy strategy can be designed to correct a speci

241 have important implications for the current gene therapy strategy for LCA that emphasizes the need f

242 ne modulation, and also opens up a promising gene therapy strategy for optic neuropathies, the most c

243 Here, we tested whether a gene therapy strategy to reduce CD33 on microglia in AD

244 KII inhibition with a cardiomyocyte-targeted gene therapy strategy would suppress arrhythmia in CPVT

245 ons Adeno-associated viral-based anti-TASK-1 gene therapy suppressed AF and corrected cellular electr

246 rapies include enzyme replacement therapies, gene therapies targeting the brain and the eye, cell the

247 8 is an excellent viral vector for inner ear gene therapy targeting cochlear hair cells and supportin

248 Gene therapy targeting secondary cone degeneration is an

249 Widespread use of gene therapy technologies is limited in part by the lack

250 We further demonstrated that gene therapy that combines AAV-mSncg promoter with clust

251 and demonstrate the potential of combination gene therapy that may improve health span and longevity

252 d10 years following AAV2-neurturin (CERE120) gene therapy, the longest post-mortem trophic factor gen

253 cell-specific exosomal delivery of drug and gene therapies to improve the functional capacity of the

254 one (SVZ) have prompted strategies targeting gene therapies to these cells to enhance neurogenesis af

255 For a genetic treatment such as gene therapy to be successful, an accurate genetic diagn

256 administration of pharmacological agents or gene therapy to further improve transplant outcomes.

257 In cardiac gene therapy to improve contractile function, achieving

258 vo AtN conversion may be a disease-modifying gene therapy to treat HD and other neurodegenerative dis

259 A compelling argument exists for gene therapy to treat this disease, as de novo protein s

260 which can potentially be used in future TCR gene-therapy to treat EBV-associated latency type II/III

261 ublic knowledge and perspectives of cell and gene therapies, to inform future research, education and

262 in vitro, including plasma from prospective gene therapy trial participants.

263 wo decades have past since the first CF lung gene therapy trials and significant advances in the ther

264 Because RPGR gene therapy trials are underway, a future imperative ex

265 Pioneering gene therapy trials have shown that the genetic engineer

266 overnment, academia, and private funding for gene therapy trials in the United States by technology t

267 The outcomes of CF lung gene therapy trials will likely inform productive paths

268 a well-characterized cohort for future TRDN gene therapy trials.

269 analysis of the emerging patent landscape of gene therapies under development, focusing on non-viral

270 Conclusion: In summary, gene therapy using an optimized therapeutic cassette in

271 ly, we have developed an effective non-viral gene therapy using self-assembled nanoparticles of a mul

272 Ex-vivo gene therapy using stem cells or T cells transduced by r

273 Adeno-associated virus (AAV) is a promising gene therapy vector because of its efficient gene delive

274 shows that targeted genomic integration of a gene therapy vector can restore the function of paternal

275 udy, we investigated the ability of KB105, a gene therapy vector encoding full-length human TGM1, to

276 ring in vivo using an adeno-associated virus gene therapy vector inhibited cardiac hypertrophy and im

277 We set out to develop an epilepsy gene therapy vector optimized for clinical translation.

278 ouse studies suggest that GAd is a promising gene therapy vector that utilizes lung ECs as a source o

279 ings underscore the potential of AAVv66 as a gene therapy vector.

280 antages with viral-based episomal-expressing gene therapy vectors include the risk of insertional mut

281 iew the challenges with surgical delivery of gene therapy vectors that limited therapeutic outcomes i

282 uses that have been developed into promising gene therapy vectors.

283 g as a scaffold for the development of human gene therapy vectors.

284 s) have recently emerged at the forefront as gene therapy vectors; however, our understanding of host

285 current products, but also emerging cell and gene therapies which have shown much therapeutic promise

286 We also discuss gene therapy, which holds promise of a cure, although it

287 linical trials, it is likely that autologous gene therapies will become standard of care for a number

288 Gene therapy with AAV5-hFVIII-SQ vector in participants

289 Gene therapy with adeno-associated virus (AAV) vectors h

290 Gene therapy with an adeno-associated vector (AAV) serot

291 Gene therapy with autologous HSCs modified to express B-

292 In previous studies, autologous gene therapy with gamma-retroviral vectors failed to rec

293 In gene therapy with human hematopoietic stem and progenito

294 is cardioprotective and suggest that cardiac gene therapy with PDE4B might constitute a new promising

295 or clinical gene therapy.IMPORTANCE Clinical gene therapy with recombinant AAV vectors has largely re

296 Gene therapy with sCX3CL1 is a promising mutation-indepe

297 utics combining the immense potential of DNA gene-therapy with the absence of genome integration-asso

298 investigate alternative approaches to brain gene therapy without viral vectors.

299 Direct in vivo gene therapy would avoid these issues, and such approach

300 An ideal tool for gene therapy would enable efficient gene integration at