Prospects for Neuroprotective Gene Therapy for Neurodegenerative Diseases

1996 ◽  
pp. 9-10
Author(s):  
Marc Peschanski
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases

scholarly journals Safety profile of the transcription factor EB (TFEB)-based gene therapy through intracranial injection in mice

2020 ◽  
Vol 11 (1) ◽  
pp. 241-250
Author(s):  
Zhenyu Li ◽  
Guangqian Ding ◽  
Yudi Wang ◽  
Zelong Zheng ◽  
Jianping Lv
Keyword(s):  
Gene Therapy ◽  
Transcription Factor ◽  
Neurodegenerative Diseases ◽  
Brain Tissue ◽  
Inflammatory Responses ◽  
Tissue Samples ◽  
Protein Levels ◽  
Virus Serotype ◽  
Transcription Factor Eb ◽  
The Brain

AbstractTranscription factor EB (TFEB)-based gene therapy is a promising therapeutic strategy in treating neurodegenerative diseases by promoting autophagy/lysosome-mediated degradation and clearance of misfolded proteins that contribute to the pathogenesis of these diseases. However, recent findings have shown that TFEB has proinflammatory properties, raising the safety concerns about its clinical application. To investigate whether TFEB induces significant inflammatory responses in the brain, male C57BL/6 mice were injected with phosphate-buffered saline (PBS), adeno-associated virus serotype 8 (AAV8) vectors overexpressing mouse TFEB (pAAV8-CMV-mTFEB), or AAV8 vectors expressing green fluorescent proteins (GFPs) in the barrel cortex. The brain tissue samples were collected at 2 months after injection. Western blotting and immunofluorescence staining showed that mTFEB protein levels were significantly increased in the brain tissue samples of mice injected with mTFEB-overexpressing vectors compared with those injected with PBS or GFP-overexpressing vectors. pAAV8-CMV-mTFEB injection resulted in significant elevations in the mRNA and protein levels of lysosomal biogenesis indicators in the brain tissue samples. No significant changes were observed in the expressions of GFAP, Iba1, and proinflammation mediators in the pAAV8-CMV-mTFEB-injected brain compared with those in the control groups. Collectively, our results suggest that AAV8 successfully mediates mTFEB overexpression in the mouse brain without inducing apparent local inflammation, supporting the safety of TFEB-based gene therapy in treating neurodegenerative diseases.

Adenovirus in the brain: recent advances of gene therapy for neurodegenerative diseases

1998 ◽  
Vol 55 (4) ◽  
pp. 333-341 ◽  
Author(s):  
M. Barkats ◽  
A. Bilang-Bleuel ◽  
M.H. Buc-Caron ◽  
M.N. Castel-Barthe ◽  
O. Corti ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases ◽  
Recent Advances ◽  
The Brain

scholarly journals Gene Therapy for Neurodegenerative Diseases

2018 ◽  
Vol 16 (1) ◽  
pp. 166-175 ◽  
Author(s):  
Vivek Sudhakar ◽  
R. Mark Richardson
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases

Clinical trial design in gene therapy for neurodegenerative diseases: Sanfilippo A syndrome

2020 ◽  
Vol 6 (5) ◽  
pp. 387396
Author(s):  
Adelaida Morte ◽  
Mariano Sust ◽  
Anna Vaque ◽  
Neus Gascon ◽  
Carlos Plata-Salaman ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Neurodegenerative Diseases ◽  
Trial Design ◽  
Clinical Trial Design

scholarly journals Gene therapy for neurodegenerative diseases: fact or fiction?

2001 ◽  
Vol 178 (5) ◽  
pp. 392-394 ◽  
Author(s):  
Janet E. Carter ◽  
Edward H. Schuchman
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases

Gene therapy for neurodegenerative diseases

2015 ◽  
Vol 21 (8) ◽  
pp. 504-512 ◽  
Author(s):  
Deirdre M. O’Connor ◽  
Nicholas M. Boulis
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases

Clinical Gene Therapy for Neurodegenerative Diseases: Past, Present, and Future

2017 ◽  
Vol 28 (11) ◽  
pp. 988-1003 ◽  
Author(s):  
Françoise Piguet ◽  
Sandro Alves ◽  
Nathalie Cartier
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases ◽  
Clinical Gene Therapy

Research Findings

2004 ◽  
Vol 08 (12) ◽  
pp. 666-669
Keyword(s):  
Gene Therapy ◽  
Neurodegenerative Diseases ◽  
Brown Rice ◽  
Genetically Engineered ◽  
Germinated Brown Rice ◽  
Novel Treatment ◽  
Research Findings ◽  
Genetically Engineered Plants ◽  
New Treatment

Pre-Germinated Brown Rice May Possibly Benefit Alzheimer’s Patients. Genetically Engineered Plants for Preventing Diabetes. Gene Therapy — A Novel Treatment for Alzheimer’s. Potential New Treatment for Stroke and Neurodegenerative Diseases.

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