Evidence map›Paper›PMID 38191664›Full record

ArticleNature biotechnology2024

Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo.

Meirui An, Aditya Raguram, Samuel W Du, Samagya Banskota, Jessie R Davis, Gregory A Newby, Paul Z Chen, Krzysztof Palczewski, David R Liu

Open access · hybridAbstract read
In one paragraph

Article in Nature biotechnology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 176 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
176citing papers in PubMed, 1 pooled it
47.2field-weighted citation impact, top 1% 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

176 citing papers in PubMed, 1 synthesis or guideline pooled it, 202 citations in OpenAlex.

  1. Pooled it
  2. Overview of Delivery Methods for Gene Editing.Methods in molecular biology (Clifton, N.J.) · 2027
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116 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

9 authors at 2 institutions in 1 country.

Meirui AnMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.ORCID http://orcid.org/0000-0002-4006-8670
Aditya RaguramMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Samuel W DuGavin Herbert Eye Institute, Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA, USA.ORCID http://orcid.org/0000-0002-9655-3659
Samagya BanskotaMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Jessie R DavisMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.
Paul Z ChenMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA.ORCID http://orcid.org/0000-0001-5261-1610
Krzysztof PalczewskiGavin Herbert Eye Institute, Center for Translational Vision Research, Department of Ophthalmology, University of California, Irvine, CA, USA.ORCID http://orcid.org/0000-0002-0788-545X
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of MIT and Harvard, Cambridge, MA, USA. drliu@fas.harvard.edu.ORCID http://orcid.org/0000-0002-9943-7557
Broad Institute · USUniversity of California, Irvine · US

Funding

Center for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7M
STRUCTURAL STUDIES OF ARRESTINSR01EY009339 · NEI · UNIVERSITY OF WASHINGTON · PI KISER, PHILIP DAVID, PALCZEWSKI, KRZYSZTOF · 1992 to 2025
$16.0M
MEDICAL SCIENTIST TRAINING PROGRAMT32GM008620 · NIGMS · UNIVERSITY OF CALIFORNIA-IRVINE · PI GOLDIN, ALAN L · 1999 to 2023
$8.1M
Integrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4M
Precision genome editing in vivo to treat retinal diseasesR01EY034501 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Audrone Lapinaite, Krzysztof Palczewski · 2023 to 2026
$3.9M
NEI UCI Center Core Grant for Vision ResearchP30EY034070 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Vladimir Jivkov Kefalov · 2022 to 2026
$3.7M
Delivery Technologies for In Vivo Genome EditingUG3AI150551 · NIAID · BETH ISRAEL DEACONESS MEDICAL CENTER · PI CHAIKOF, ELLIOT · 2019 to 2021
$2.3M
Expanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1M
microRNA-204 and microRNA-211 regulation of RPE phagocytosisF30EY033642 · NEI · UNIVERSITY OF CALIFORNIA-IRVINE · PI Samuel Wang Du · 2022 to 2026
$241k
Howard Hughes Medical Institute (HHMI) Liu InvestigatorshipNEI NIH HHS F30 EY033642NEI NIH HHS P30 EY034070NEI NIH HHS R01 EY009339NEI NIH HHS R01 EY034501NHGRI NIH HHS RM1 HG009490NIAID NIH HHS U01 AI142756NIAID NIH HHS UG3 AI150551NIGMS NIH HHS R35 GM118062NIGMS NIH HHS T32 GM008620U.S. Department of Health & Human Services | NIH | National Eye Institute (NEI) R01EY009339U.S. Department of Health & Human Services | NIH | National Human Genome Research Institute (NHGRI) RM1HG009490U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases (NIAID) U01AI142756U.S. Department of Health & Human Services | NIH | National Institute of Allergy and Infectious Diseases (NIAID) UG3AI150551U.S. Department of Health & Human Services | NIH | National Institute of General Medical Sciences (NIGMS) R35GM118062
6 · The paper itself

Abstract

Prime editing enables precise installation of genomic substitutions, insertions and deletions in living systems. Efficient in vitro and in vivo delivery of prime editing components, however, remains a challenge. Here we report prime editor engineered virus-like particles (PE-eVLPs) that deliver prime editor proteins, prime editing guide RNAs and nicking single guide RNAs as transient ribonucleoprotein complexes. We systematically engineered v3 and v3b PE-eVLPs with 65- to 170-fold higher editing efficiency in human cells compared to a PE-eVLP construct based on our previously reported base editor eVLP architecture. In two mouse models of genetic blindness, single injections of v3 PE-eVLPs resulted in therapeutically relevant levels of prime editing in the retina, protein expression restoration and partial visual function rescue. Optimized PE-eVLPs support transient in vivo delivery of prime editor ribonucleoproteins, enhancing the potential safety of prime editing by reducing off-target editing and obviating the possibility of oncogenic transgene integration.

Indexed as

Gene EditingRibonucleoproteinsAnimalsHumansMiceRNA, Guide, CRISPR-Cas SystemsVirionRibonucleoproteinsRNA, Guide, CRISPR-Cas Systems

Identifiers

PMID38191664
PMCPMC11228131
OpenAlexW4390670214

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.