ライフサイエンスコーパス: genetic therapy

1 akes the hammerhead ribozyme a candidate for genetic therapy.

2 nts are suitable candidates for this type of genetic therapy.

3 oach might lead to new types of vaccines and genetic therapies.

4 help to enhance the efficacy of virus-based genetic therapies.

5 nd types of genetic cargo to develop diverse genetic therapies.

6 ng of RDEB patients, while awaiting curative genetic therapies.

7 ression, making them an important target for genetic therapies.

8 chnology could be applied to a wide range of genetic therapies.

9 iness among individuals with SCD considering genetic therapies.

10 nterventions such as stem cell and molecular genetic therapies.

11 d disorders and spurring renewed interest in genetic therapies.

12 es an opportunity to interfere with HL using genetic therapies.

13 s on the current advances and limitations in genetic therapies against HIV, including the status of s

14 Ribozymes are promising genetic therapy agents and should, in the future, play a

15 In this study, we assessed two different genetic therapies-an adenine base editor (ABE8e) and a p

16 and clinical ecosystems enabling customized genetic therapies and explores the innovation, framework

17 facile treatments, such as in-vivo-delivered genetic therapies and new drugs that can eventually be a

18 Skin is an attractive target for delivery of genetic therapies and vaccines.

19 hnological advances promise to revolutionize genetic therapy and, as I discuss, represent the culmina

20 N-of-1 individualized therapies, focusing on genetic therapies, and illustrate advances and challenge

21 ein and peptide therapy, cell-based therapy, genetic therapy, application of scaffolds, bone anabolic

22 In recent years, genetic therapy approaches have been explored in preclin

23 No specific medical or genetic therapies are available for any type of EDS.

24 Novel therapeutic compounds and several genetic therapies are currently under investigation, but

25 Although genetic therapies are maturing as the rules of bioenerge

26 Directed cytokine and genetic therapies are on the horizon.

27 CRISPR genetic therapies are revolutionizing the landscape of p

28 At the same time, such genetic therapies are the most expensive drugs on the ma

29 t the long-term safety and efficacy of these genetic therapies are yet to be ascertained.

30 The genetic therapy as well as treatment with dendrimer alon

31 of human multistep carcinogenesis, validated genetic therapy by 3-phosphoinositide-dependent protein

32 Drug and genetic therapies can now be tested in this mouse model

33 This suggests that future genetic therapies could address both muscle and brain dy

34 Given that genetic therapies currently tested in HD are primarily e

35 beta-thalassemia, raising the potential for genetic therapy directed at enhancing HbF.

36 During the past 10years, genetic therapy directed toward correction of RNA mis-sp

37 The genetic therapy exploited a truncated HIF-1alpha protein

38 ults indicate that the therapeutic window of genetic therapies for Angelman syndrome is broader than

39 the feasibility of targeting platelets with genetic therapies for better management of patients with

40 cell development and for the development of genetic therapies for diseases involving hematopoietic c

41 luence the coagulation system, the future of genetic therapies for hemostasis is bright.

42 stem will prove useful for the evaluation of genetic therapies for hFVIII immunomodulation and bring

43 rapies for hFVIII immunomodulation and bring genetic therapies for hFVIII tolerance closer to clinica

44 tions that successful clinical transition of genetic therapies for PMDs is feasible.

45 asis in disease, and testing of cellular and genetic therapies for the correction of thalassemia.

46 es could have implications for the design of genetic therapies for treatment of Fanconi anemia and po

47 egulatory protein Rev has been explored as a genetic therapy for AIDS.

48 orneal limbus may provide a feasible mode of genetic therapy for corneal disorders with an epithelial

49 n future studies concerning the potential of genetic therapy for DMD, as well as other muscle disorde

50 r and expression suggests the feasibility of genetic therapy for retinal disease.

51 The main obstacle to genetic therapies has been the development of vectors ab

52 Genetic therapies have attempted introduction of nucleic

53 Twenty genetic therapies have been approved by the US Food and

54 and myotonic dystrophy (DM), but attempts at genetic therapy have yet to prove successful.

55 Monogenetic diseases are ideal targets for genetic therapies; however, the blood-brain barrier (BBB

56 Review, we describe the current landscape of genetic therapies in ALS, highlighting achievements and

57 strategies are currently at the forefront of genetic therapies in CVD, with several ribonucleic acid-

58 nant retroviral vectors to effect corrective genetic therapies in hematopoietic stem cells (HSCs) has

59 er, obstacles to the clinical application of genetic therapies in PMDs remain; the development of inn

60 n, will be crucial for the future success of genetic therapies in the brain.

61 dverse events are an inherent risk of CRISPR genetic therapies, including off-target edits.

62 s not harmful and allowing possibilities for genetic therapy interventions that utilize overexpressio

63 Genetic therapy is undergoing a renaissance with expansi

64 studies support a potential that single-dose genetic therapies may be through the correction of patho

65 Individualized genetic therapies-medicines that precisely target a gene

66 l vectors has been particularly critical for genetic therapies of hematological diseases.

67 riction endonucleases are feasible tools for genetic therapy of a sub-group of mitochondrial disorder

68 Lessons learned from systemic genetic therapy of muscle disorders also should have imp

69 Stem cell transplantation and genetic therapies offer potential cures for patients wit

70 Although genetic therapies offer the ability to modulate neuronal

71 Inhaled genetic therapies offer the prospect of addressing the u

72 Genetic therapies, particularly adeno-associated viruses

73 Personalized genetic therapy represents one promising approach for th

74 The systemic delivery of genetic therapies required for the treatment of inaccess

75 use models exhibit similar phenotypes, where genetic therapies restoring brain dystrophins improve th

76 g genetic variants and in the development of genetic therapy strategies present exciting new therapeu

77 Based on these results, genetic therapies targeted towards selective manipulatio

78 We identified small molecule and genetic therapies that normalized the phenotype of NAA10

79 eroid treatment improves outcomes and on new genetic therapies that require early diagnosis for effec

80 c basis of the syndromes is hoped to lead to genetic therapy that can restore repolarisation.

81 ghlights genome editing as compared to other genetic therapies, the differences between editing strat

82 hold promise for the clinical application of genetic therapies to combat AIDS.

83 trate considerable potential for single-dose genetic therapies to correct or silence pathogenic varia

84 recent progress toward the establishment of genetic therapies to treat inherited neuromuscular disor

85 n to develop persistent, effective non-viral genetic therapies to treat this condition.

86 met the inclusion criteria for a current ALS genetic-therapy trial.

87 Current strategies for genetic therapy using Moloney retroviruses require ex vi

88 harnessed for the targeted delivery of human genetic therapies via the introduction of exogenous gene

89 One emerging avenue is genetic therapies, which hold particular promise for dis

90 For many of them, genetic therapies will be tested in this or the coming y

91 ealth care and the increasing feasibility of genetic therapy will, although slowly, augment the futur