Evidence map›Paper›PMID 30706401›Full record

ArticleMethods in molecular biology (Clifton, N.J.)2019

Co-Delivery of a Short-Hairpin RNA and a shRNA-Resistant Replacement Gene with Adeno-Associated Virus: An Allele-Independent Strategy for Autosomal-Dominant Retinal Disorders.

Michael T Massengill, Brianna M Young, Alfred S Lewin, Cristhian J Ildefonso

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Methods in molecular biology (Clifton, N.J.), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
3.0field-weighted citation impact, top 7% 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

7 citing papers in PubMed, 9 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Review
  7. Short Hairpin RNAs for Strand-Specific Small Interfering RNA Production.Frontiers in bioengineering and biotechnology · 2020
    Article
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 2 institutions in 1 country.

Michael T MassengillDepartment of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA.
Brianna M YoungDepartment of Ophthalmology, University of Florida College of Medicine, Gainesville, FL, USA.
Alfred S LewinDepartment of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA.
Cristhian J IldefonsoDepartment of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA. ildefons@ufl.edu.
Florida College · USUniversity of Florida · US

Funding

Translational Gene Therapy for Rhodopsin Autosomal Dominant Retinitis PigmentosaR24EY022012 · NEI · UNIVERSITY OF PENNSYLVANIA · PI BELTRAN, WILLIAM A. · 2012 to 2016
$7.9M
Testing Gene Therapy in Models of Geographic AtrophyR01EY026268 · NEI · UNIVERSITY OF FLORIDA · PI LEWIN, ALFRED S · 2016 to 2020
$2.6M
TX OF AUTOSOMAL DOMINANT EYE DISEASE USING CATALYTIC RNAR01EY011596 · NEI · UNIVERSITY OF FLORIDA · PI LEWIN, ALFRED S · 1997 to 2005
$1.9M
Development of Allele Independent Gene Therapy Strategies for Autosomal Dominant Retinitis PigmentsaF30EY027163 · NEI · UNIVERSITY OF FLORIDA · PI MASSENGILL, MICHAEL · 2016 to 2019
$177k
NEI NIH HHS F30 EY027163NEI NIH HHS R01 EY011596NEI NIH HHS R01 EY026268
6 · The paper itself

Abstract

Recombinant adeno-associated virus (rAAV) has become an important gene delivery vector for the treatment of inherited retinal degenerative diseases. Many of the mutations leading to retinal degeneration are inherited in an autosomal-dominant pattern and can produce toxic gain-of-function and/or dominant-negative effects. Here we describe an allele-independent gene therapy strategy with rAAV to treat autosomal-dominant retinal degenerative diseases. In this methodology, we co-deliver a short-hairpin RNA (shRNA) to inhibit expression of both the toxic and (WT) copies of the gene as well as an shRNA-resistant cDNA for functional gene replacement with a rAAV.

Indexed as

AnimalsDependovirusDisease Models, AnimalGenetic TherapyGenetic VectorsHEK293 CellsHumansInjections, IntraocularMiceRetinal DegenerationRNA, Small InterferingRNA, Small InterferingAllele-independentAutosomal-dominantGene therapyRecombinant adeno-associated virusRetinal degenerationShort-hairpin RNAshRNA-resistant cDNA

Identifiers

PMID30706401
PMCPMC6613586
OpenAlexW2914748261

What OpenQuestion holds

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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.