Targeted Delivery for Neurodegenerative Disorders Using Gene Therapy Vectors: Gene Next Therapeutic Goals

被引：5

作者：

Singh, Manisha ^{[1
]}

Singh, Surinder P. ^{[2
]}

Yadav, Deepshikha ^{[2
,3
,4
]}

Agarwal, Mugdha ^{[1
]}

Agarwal, Shriya ^{[1
]}

Agarwal, Vinayak ^{[1
]}

Swargiary, Geeta ^{[1
]}

Srivastava, Sahil ^{[1
]}

Tyagi, Sakshi ^{[1
]}

Kaur, Ramneek ^{[5
]}

Mani, Shalini ^{[1
]}

机构：

[1] Jaypee Inst Informat Technol JILT Noida, Dept Biotechnol, Noida, UP, India

[2] CSIR Natl Phys Lab, Bhartiya Nirdeshak Dravya Div, New Delhi, India

[3] CSIR Natl Phys Lab, Physicomech Metrol Div, New Delhi, India

[4] Acad Sci & Innovat Res AcSIR, Ghaziabad, UP, India

[5] Western Sydney Univ, Sch Med, Campbelltown, NSW, Australia

来源：

CURRENT GENE THERAPY | 2021年 / 21卷 / 01期

关键词：

Gene therapy; neurodegenerative diseases; viral vectors; nonviral vectors; Alzheimer disease; Parkinson disease; schizophrenia retroviruses; SPINAL-MUSCULAR-ATROPHY; SURVIVAL-MOTOR-NEURON; AMYOTROPHIC-LATERAL-SCLEROSIS; MOUSE MODEL; PARKINSONS-DISEASE; IN-VIVO; ALZHEIMERS-DISEASE; PROLONGS SURVIVAL; MULTIPLE-SCLEROSIS; MUTANT HUNTINGTIN;

D O I：

10.2174/1566523220999200817164907

中图分类号：

Q3 [遗传学];

学科分类号：

071007 ; 090102 ;

摘要：

The technique of gene therapy, ever since its advent nearly fifty years ago, has been utilized by scientists as a potential treatment option for various disorders. This review discusses some of the major neurodegenerative diseases (NDDs) like Alzheimer's disease (AD), Parkinson's Disease (PD), Motor neuron diseases (MND), Spinal Muscular Atrophy (SMA), Huntington's Disease (HD), Multiple Sclerosis (MS), etc. and their underlying genetic mechanisms along with the role that gene therapy can play in combating them. The pathogenesis and the molecular mechanisms specifying the altered gene expression of each of these NDDs have also been discussed in elaboration. The use of gene therapy vectors can prove to be an effective tool in the field of curative modern medicine for the generations to come. Therefore, consistent efforts and progressive research towards its implementation can provide us with powerful treatment options for disease conditions that have so far been considered as incurable.

引用

页码：23 / 42

页数：20

共 50 条

[1] The therapeutic potential of siRNA in gene therapy of neurodegenerative disorders
Koutsilieri, E.
Rethwilm, A.
Scheller, C.
JOURNAL OF NEURAL TRANSMISSION-SUPPLEMENT, 2007, (72): : 43 - 49
[2] Targeted delivery of functional genes for gene therapy using carbohydrates containing vectors
Krivtsov, GG
Zhdanov, RI
VOPROSY MEDITSINSKOI KHIMII, 2000, 46 (03): : 246 - 255
[3] Adeno-associated virus vectors for gene therapy of neurodegenerative disorders
During, MJ
Leone, P
CLINICAL NEUROSCIENCE, 1996, 3 (05) : 292 - 300
[4] Emerging Perspectives on Gene Therapy Delivery for Neurodegenerative and Neuromuscular Disorders
Limia, Cintia Gomez
Baird, Megan
Schwartz, Maura
Saxena, Smita
Meyer, Kathrin
Wein, Nicolas
JOURNAL OF PERSONALIZED MEDICINE, 2022, 12 (12):
[5] Selection of Next Generation AAV Gene Therapy Vectors for Specific and Precise Gene Delivery
Muench, Robert
Kay, Mark
MOLECULAR THERAPY, 2016, 24 : S101 - S101
[6] Therapeutic globin gene delivery using lentiviral vectors
Rivella, S
Sadelain, M
CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2002, 4 (05) : 505 - 514
[7] Renal targeted gene delivery vectors
Denby, Laura
Work, Lorraine M.
Von Seggern, Dan J.
Wu, Eugene
Hunt, Ela
Nicklin, Stuart A.
Baker, Andrew H.
JOURNAL OF HYPERTENSION, 2006, 24 : 179 - 179
[8] Targeted synthetic gene delivery vectors
Schaffer, DV
Lauffenburger, DA
CURRENT OPINION IN MOLECULAR THERAPEUTICS, 2000, 2 (02) : 155 - 161
[9] Therapeutic delivery issues in gene therapy, part 1: Vectors
Parasrampuria, DA
BIOPHARM-THE APPLIED TECHNOLOGIES OF BIOPHARMACEUTICAL DEVELOPMENT, 1998, 11 (03): : 38 - +
[10] Gene therapy for neurodegenerative and ocular diseases using lentiviral vectors
Ralph, GS
Binley, K
Wong, LF
Azzouz, M
Mazarakis, ND
CLINICAL SCIENCE, 2006, 110 (01) : 37 - 46

← 1 2 3 4 5 →