Evidence map›Paper›PMID 33998992›Full record

ReviewCurrent neuropharmacology2021

Cell-Based Delivery Approaches for DNA-Binding Domains to the Central Nervous System.

Peter Deng, Julian Halmai, Jennifer J Waldo, Kyle D Fink

Open access · bronzeAbstract readReview
In one paragraph

Review in Current neuropharmacology, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
0.1field-weighted citation impact, top 57% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed, 1 citations in OpenAlex.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

4 authors at 1 institution in 1 country.

Peter DengDepartment of Neurology, Stem Cell Program and Gene Therapy Center, UC Davis Medical Center, Sacramento, CA, United States.
Julian HalmaiDepartment of Neurology, Stem Cell Program and Gene Therapy Center, UC Davis Medical Center, Sacramento, CA, United States.
Jennifer J WaldoDepartment of Neurology, Stem Cell Program and Gene Therapy Center, UC Davis Medical Center, Sacramento, CA, United States.
Kyle D FinkDepartment of Neurology, Stem Cell Program and Gene Therapy Center, UC Davis Medical Center, Sacramento, CA, United States.
University of California Davis Medical Center · US

Funding

UC Davis Clinical and Translational Science CenterUL1TR001860 · NCATS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI KENYON, NICHOLAS J., LYLES, COURTNEY REES · 2016 to 2025
$47.3M
Predoctoral Training in Pharmacological SciencesT32GM099608 · NIGMS · UNIVERSITY OF CALIFORNIA AT DAVIS · PI HELL, JOHANNES W · 2012 to 2021
$2.2M
California Institute for Regenerative Medicine Discovery DISC2-09032. R24, 3R24OD021606-02S1National Center for Advancing Translational Sciences, National Institutes of Health UL1 TR001860National Institute for General Medical Sciences Pharmacology T32 GM099608
6 · The paper itself

Abstract

Advancements in programmable DNA-Binding Proteins (DBDs) that target the genome, such as zinc fingers, transcription activator-like effectors, and Cas9, have broadened drug target design beyond traditional protein substrates. Effective delivery methodologies remain a major barrier in targeting the central nervous system. Currently, adeno-associated virus is the most wellvalidated delivery system for the delivery of DBDs towards the central nervous with multiple, ongoing clinical trials. While effective in transducing neuronal cells, viral delivery systems for DBDs remain problematic due to inherent viral packaging limits or immune responses that hinder translational potential. Direct administration of DBDs or encapsulation in lipid nanoparticles may provide alternative means towards delivering gene therapies into the central nervous system. This review will evaluate the strengths and limitations of current DBD delivery strategies in vivo. Furthermore, this review will discuss the use of adult stem cells as a putative delivery vehicle for DBDs and the potential advantages that these systems have over previous methodologies.

Indexed as

Gene EditingNanoparticlesCentral Nervous SystemDNALiposomesDNALipid NanoparticlesLiposomesAngelman syndromeartificial transcription factorsCRISPR/Cas9DNA-binding domainsgene therapy.lipid nanoparticlemesenchymal stem cellzinc finger

Identifiers

PMID33998992
PMCPMC9185769
OpenAlexW3161595907

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.