Evidence map›Paper›PMID 32069700›Full record

ArticleMolecular therapy. Nucleic acids2020

Development of a Facile Approach for Generating Chemically Modified CRISPR/Cas9 RNA.

Tristan Scott, Citradewi Soemardy, Kevin V Morris

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Nucleic acids, 2020. 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
0.5field-weighted citation impact, top 38% 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, 7 citations in OpenAlex.

  1. Article
  2. Delivery of gene editing therapeutics.Nanomedicine : nanotechnology, biology, and medicine · 2023
    Review
  3. Gene Editing in Hematopoietic Stem Cells.Advances in experimental medicine and biology · 2023
    Review
  4. Article
  5. Review
  6. Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Review
  7. Review
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

3 authors at 2 institutions in 1 country.

Tristan ScottCenter for Gene Therapy, City of Hope-Beckman Research Institute and Hematological Malignancy and Stem Cell Transplantation Institute at the City of Hope, 1500 E. Duarte Road, Duarte, CA 91010, USA.
Citradewi SoemardyCenter for Gene Therapy, City of Hope-Beckman Research Institute and Hematological Malignancy and Stem Cell Transplantation Institute at the City of Hope, 1500 E. Duarte Road, Duarte, CA 91010, USA.
Kevin V MorrisCenter for Gene Therapy, City of Hope-Beckman Research Institute and Hematological Malignancy and Stem Cell Transplantation Institute at the City of Hope, 1500 E. Duarte Road, Duarte, CA 91010, USA. Electronic address: kmorris@coh.org.
Beckman Research InstituteCity of Hope · US

Funding

Transcriptional control, targeted modification, and excision of HIV in the brainR01MH113407 · NIMH · BECKMAN RESEARCH INSTITUTE/CITY OF HOPE · PI BURNETT, JOHN CORY · 2017 to 2021
$3.0M
Targeted inhibition of HIV-1 latencyR01AI111139 · NIAID · SCRIPPS RESEARCH INSTITUTE, THE · PI MORRIS, KEVIN V · 2014 to 2018
$2.7M
Activating cystic fibrosis transmembrane conductance regulator: the therapeutic potential of RNA directed gene activationR01DK104681 · NIDDK · SCRIPPS RESEARCH INSTITUTE, THE · PI MORRIS, KEVIN V · 2015 to 2019
$1.9M
NIAID NIH HHS R01 AI111139NIDDK NIH HHS R01 DK104681NIMH NIH HHS R01 MH113407
6 · The paper itself

Abstract

The RNA-guided, modified type II prokaryotic CRISPR with CRISPR-associated proteins (CRISPR/Cas9) system represents a simple gene-editing platform with applications in biotechnology and also potentially as a therapeutic modality. The system requires a small guide RNA (sgRNA) and a catalytic Cas9 protein to induce non-homologous end joining (NHEJ) at break sites, resulting in the formation of inactivating mutations, or through homology-directed repair (HDR) can engineer in specific sequence changes. Although CRISPR/Cas9 is a powerful technology, the effects can be limited as a result of nuclease-mediated degradation of the RNA components. Significant research has focused on the solid-phase synthesis of CRISPR RNA components with chemically modified bases, but this approach is technically challenging and expensive. Development of a simple, generic approach to generate chemically modified CRISPR RNAs may broaden applications that require nuclease-resistant CRISPR components. We report here the development of a novel, functional U-replaced trans-activating RNA (tracrRNA) that can be in vitro transcribed with chemically stabilizing 2'-fluoro (2'F)-pyrimidines. These data represent a unique and facile approach to generating chemically stabilized CRISPR RNA.

Identifiers

PMID32069700
PMCPMC7019045
OpenAlexW3000091413

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.