Evidence map›Paper›PMID 33484965›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2021

A versatile platform for generating engineered extracellular vesicles with defined therapeutic properties.

Kevin Dooley, Russell E McConnell, Ke Xu, Nuruddeen D Lewis, Sonya Haupt, Madeleine R Youniss, Shelly Martin, Chang Ling Sia, Christine McCoy, Raymond J Moniz and 18 more

Abstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 191 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
191citing 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

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

  1. Pooled it
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  16. Endogenous Engineering Reprograms Extracellular Vesicles for Enhanced Therapeutic Function.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026
    Review
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131 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

28 authors.

Kevin DooleyCodiak BioSciences, Cambridge, MA 02140, USA. Electronic address: kevin.dooley@codiakbio.com.
Russell E McConnellCodiak BioSciences, Cambridge, MA 02140, USA.
Ke XuCodiak BioSciences, Cambridge, MA 02140, USA.
Nuruddeen D LewisCodiak BioSciences, Cambridge, MA 02140, USA.
Sonya HauptCodiak BioSciences, Cambridge, MA 02140, USA.
Madeleine R YounissCodiak BioSciences, Cambridge, MA 02140, USA.
Shelly MartinCodiak BioSciences, Cambridge, MA 02140, USA.
Chang Ling SiaCodiak BioSciences, Cambridge, MA 02140, USA.
Christine McCoyCodiak BioSciences, Cambridge, MA 02140, USA.
Raymond J MonizCodiak BioSciences, Cambridge, MA 02140, USA.
Olga BurenkovaCodiak BioSciences, Cambridge, MA 02140, USA.
Jorge Sanchez-SalazarCodiak BioSciences, Cambridge, MA 02140, USA.
Su Chul JangCodiak BioSciences, Cambridge, MA 02140, USA.
Bryan ChoiCodiak BioSciences, Cambridge, MA 02140, USA.
Rane A HarrisonCodiak BioSciences, Cambridge, MA 02140, USA.
Damian HoudeCodiak BioSciences, Cambridge, MA 02140, USA.
Dalia BurzynCodiak BioSciences, Cambridge, MA 02140, USA.
Charan LengCodiak BioSciences, Cambridge, MA 02140, USA.
Katherine KirwinCodiak BioSciences, Cambridge, MA 02140, USA.
Nikki L RossCodiak BioSciences, Cambridge, MA 02140, USA.
Jonathan D FinnCodiak BioSciences, Cambridge, MA 02140, USA.
Leonid GaidukovCodiak BioSciences, Cambridge, MA 02140, USA.
Kyriakos D EconomidesCodiak BioSciences, Cambridge, MA 02140, USA.
Scott EstesCodiak BioSciences, Cambridge, MA 02140, USA.
James E ThorntonCodiak BioSciences, Cambridge, MA 02140, USA.
John D KulmanCodiak BioSciences, Cambridge, MA 02140, USA.
Sriram SathyanarayananCodiak BioSciences, Cambridge, MA 02140, USA.
Douglas E WilliamsCodiak BioSciences, Cambridge, MA 02140, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Extracellular vesicles (EVs) are an important intercellular communication system facilitating the transfer of macromolecules between cells. Delivery of exogenous cargo tethered to the EV surface or packaged inside the lumen are key strategies for generating therapeutic EVs. We identified two "scaffold" proteins, PTGFRN and BASP1, that are preferentially sorted into EVs and enable high-density surface display and luminal loading of a wide range of molecules, including cytokines, antibody fragments, RNA binding proteins, vaccine antigens, Cas9, and members of the TNF superfamily. Molecules were loaded into EVs at high density and exhibited potent in vitro activity when fused to full-length or truncated forms of PTGFRN or BASP1. Furthermore, these engineered EVs retained pharmacodynamic activity in a variety of animal models. This engineering platform provides a simple approach to functionalize EVs with topologically diverse macromolecules and represents a significant advance toward unlocking the therapeutic potential of EVs.

Indexed as

AnimalsCell CommunicationDrug Delivery SystemsExtracellular VesiclesFemaleHEK293 CellsHumansMembrane ProteinsMiceNeoplasm ProteinsNerve Tissue ProteinsProteinsRepressor ProteinsBASP1 protein, humanMembrane ProteinsNeoplasm ProteinsNerve Tissue ProteinsProteinsPTGFRN protein, humanRepressor ProteinsBASP1EV engineeringexosome engineeringexosomesextracellular vesiclesIL-12PTGFRNsacffoldvaccine

Identifiers

PMID33484965
PMCPMC8116569

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.