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Gene Therapy for the Nervous System: Challenges and New Strategies

Overview of attention for article published in Neurotherapeutics, October 2014
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (90th percentile)
  • High Attention Score compared to outputs of the same age and source (83rd percentile)

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1 blog
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5 X users
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66 patents

Citations

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70 Dimensions

Readers on

mendeley
189 Mendeley
Title
Gene Therapy for the Nervous System: Challenges and New Strategies
Published in
Neurotherapeutics, October 2014
DOI 10.1007/s13311-014-0299-5
Pubmed ID
Authors

Casey A. Maguire, Servio H. Ramirez, Steven F. Merkel, Miguel Sena-Esteves, Xandra O. Breakefield

Abstract

Current clinical treatments for central nervous system (CNS) diseases, such as Parkinson's disease and glioblastoma do not halt disease progression and have significant treatment morbidities. Gene therapy has the potential to "permanently" correct disease by bringing in a normal gene to correct a mutant gene deficiency, knocking down mRNA of mutant alleles, and inducing cell-death in cancer cells using transgenes encoding apoptosis-inducing proteins. Promising results in clinical trials of eye disease (Leber's congenital aumorosis) and Parkinson's disease have shown that gene-based neurotherapeutics have great potential. The recent development of genome editing technology, such as zinc finger nucleases, TALENS, and CRISPR, has made the ultimate goal of gene correction a step closer. This review summarizes the challenges faced by gene-based neurotherapeutics and the current and recent strategies designed to overcome these barriers. We have chosen the following challenges to focus on in this review: (1) delivery vehicles (both virus and nonviral), (2) use of promoters for vector-mediated gene expression in CNS, and (3) delivery across the blood-brain barrier. The final section (4) focuses on promising pre-clinical/clinical studies of neurotherapeutics.

X Demographics

X Demographics

The data shown below were collected from the profiles of 5 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 189 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
United States 2 1%
United Kingdom 1 <1%
Denmark 1 <1%
Australia 1 <1%
Unknown 184 97%

Demographic breakdown

Readers by professional status Count As %
Researcher 31 16%
Student > Bachelor 31 16%
Student > Ph. D. Student 28 15%
Student > Master 22 12%
Student > Doctoral Student 12 6%
Other 34 18%
Unknown 31 16%
Readers by discipline Count As %
Agricultural and Biological Sciences 40 21%
Biochemistry, Genetics and Molecular Biology 35 19%
Neuroscience 27 14%
Medicine and Dentistry 23 12%
Pharmacology, Toxicology and Pharmaceutical Science 8 4%
Other 19 10%
Unknown 37 20%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 16. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 05 March 2024.
All research outputs
#2,276,816
of 25,374,647 outputs
Outputs from Neurotherapeutics
#201
of 1,307 outputs
Outputs of similar age
#24,960
of 265,645 outputs
Outputs of similar age from Neurotherapeutics
#2
of 12 outputs
Altmetric has tracked 25,374,647 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 91st percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 1,307 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 18.2. This one has done well, scoring higher than 84% of its peers.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 265,645 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 90% of its contemporaries.
We're also able to compare this research output to 12 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 83% of its contemporaries.