Evidence map›Paper›PMID 36293232›Full record

ReviewInternational journal of molecular sciences2022

Base and Prime Editing in the Retina-From Preclinical Research toward Human Clinical Trials.

Tiffany Yee, Katherine J Wert

Open access · goldAbstract readReview
In one paragraph

Review in International journal of molecular sciences, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 papers.

0numbers the graph read from it
0cells of the map it votes in
10citing papers in PubMed
1.8field-weighted citation impact, top 14% 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

10 citing papers in PubMed, 20 citations in OpenAlex.

  1. Review
  2. Review
  3. Article
  4. Review
  5. Article
  6. Review
  7. Article
  8. Review
  9. Counteracting the Common Shwachman-Diamond Syndrome-CausingInternational journal of molecular sciences · 2023
    Article
  10. 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

2 authors at 1 institution in 1 country.

Tiffany YeeDepartment of Ophthalmology, UT Southwestern Medical Center, Dallas, TX 75390, USA.ORCID 0000-0002-8709-8017
Katherine J WertDepartment of Ophthalmology, UT Southwestern Medical Center, Dallas, TX 75390, USA.
Southwestern Medical Center · US

Funding

Stem Cell, Organoid and Cell Phenotyping ModuleP30EY030413 · NEI · UT SOUTHWESTERN MEDICAL CENTER · PI W MATTHEW PETROLL · 2019 to 2026
$5.9M
Mechanisms of Disease and Translational ScienceT32GM131945 · NIGMS · UT SOUTHWESTERN MEDICAL CENTER · PI Thomas Joseph Carroll · 2019 to 2026
$1.7M
Hamon Center for Regenerative Science and Medicine Fellowship N/ANEI NIH HHS P30 EY030413NEI NIH HHS P30EY030413NIGMS NIH HHS T32 GM131945NIH HHS 5T32GM131945Van Sickle Family Foundation Award N/A
6 · The paper itself

Abstract

Inherited retinal diseases (IRDs) are a clinically and genetically heterogeneous group of diseases that are one of the leading causes of vision loss in young and aged individuals. IRDs are mainly caused by a loss of the post-mitotic photoreceptor neurons of the retina, or by the degeneration of the retinal pigment epithelium. Unfortunately, once these cells are damaged, it is irreversible and leads to permanent vision impairment. Thought to be previously incurable, gene therapy has been rapidly evolving to be a potential treatment to prevent further degeneration of the retina and preserve visual function. The development of clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9) base and prime editors have increased the capabilities of the genome editing toolbox in recent years. Both base and prime editors evade the creation of double-stranded breaks in deoxyribonucleic acid (DNA) and the requirement of donor template of DNA for repair, which make them advantageous methods in developing clinical therapies. In addition, establishing a permanent edit within the genome could be better suited for patients with progressive degeneration. In this review, we will summarize published uses of successful base and prime editing in treating IRDs.

Indexed as

CRISPR-Cas SystemsRetinal DiseasesAgedCRISPR-Associated Protein 9DNAGene EditingHumansRetinaCRISPR-Associated Protein 9DNAAAVCRISPRgene editinggene therapyphotoreceptor degenerationretinaretinal degeneration

Identifiers

PMID36293232
PMCPMC9604474
OpenAlexW4306385458

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.