Evidence map›Paper›PMID 29801422›Full record

ReviewDrug delivery2018

Delivering CRISPR: a review of the challenges and approaches.

Christopher A Lino, Jason C Harper, James P Carney, Jerilyn A Timlin

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Drug delivery, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 551 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
551citing papers in PubMed, 1 pooled it
–field-weighted citation impact
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

551 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. CRISPR/Cas Systems: Biological Basis and Genome Editing Applications.Methods in molecular biology (Clifton, N.J.) · 2027
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491 more citing papers are in PubMed but not listed here.

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.

Christopher A Linoa Bioenergy and Defense Technologies, Sandia National Laboratories , Albuquerque , NM , USA.
Jason C Harpera Bioenergy and Defense Technologies, Sandia National Laboratories , Albuquerque , NM , USA.
James P Carneya Bioenergy and Defense Technologies, Sandia National Laboratories , Albuquerque , NM , USA.
Jerilyn A Timlina Bioenergy and Defense Technologies, Sandia National Laboratories , Albuquerque , NM , USA.ORCID http://orcid.org/0000-0003-2953-1721

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Gene therapy has long held promise to correct a variety of human diseases and defects. Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the mechanism of the CRISPR-based prokaryotic adaptive immune system (CRISPR-associated system, Cas), and its repurposing into a potent gene editing tool has revolutionized the field of molecular biology and generated excitement for new and improved gene therapies. Additionally, the simplicity and flexibility of the CRISPR/Cas9 site-specific nuclease system has led to its widespread use in many biological research areas including development of model cell lines, discovering mechanisms of disease, identifying disease targets, development of transgene animals and plants, and transcriptional modulation. In this review, we present the brief history and basic mechanisms of the CRISPR/Cas9 system and its predecessors (ZFNs and TALENs), lessons learned from past human gene therapy efforts, and recent modifications of CRISPR/Cas9 to provide functions beyond gene editing. We introduce several factors that influence CRISPR/Cas9 efficacy which must be addressed before effective in vivo human gene therapy can be realized. The focus then turns to the most difficult barrier to potential in vivo use of CRISPR/Cas9, delivery. We detail the various cargos and delivery vehicles reported for CRISPR/Cas9, including physical delivery methods (e.g. microinjection; electroporation), viral delivery methods (e.g. adeno-associated virus (AAV); full-sized adenovirus and lentivirus), and non-viral delivery methods (e.g. liposomes; polyplexes; gold particles), and discuss their relative merits. We also examine several technologies that, while not currently reported for CRISPR/Cas9 delivery, appear to have promise in this field. The therapeutic potential of CRISPR/Cas9 is vast and will only increase as the technology and its delivery improves.

Indexed as

AnimalsClustered Regularly Interspaced Short Palindromic RepeatsGene EditingGenetic TherapyGene Transfer TechniquesHumansCas9CRISPRdrug deliveryhistoryoverviewprospectivereview

Identifiers

PMID29801422
PMCPMC6058482

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.