Evidence map›Paper›PMID 36896650›Full record

ReviewExpert opinion on drug delivery2023

Shaping the future from the small scale: dry powder inhalation of CRISPR-Cas9 lipid nanoparticles for the treatment of lung diseases.

Simone P Carneiro, Antonietta Greco, Enrica Chiesa, Ida Genta, Olivia M Merkel

Open access · hybridAbstract readReview
In one paragraph

Review in Expert opinion on drug delivery, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.

0numbers the graph read from it
0cells of the map it votes in
11citing papers in PubMed
4.0field-weighted citation impact, top 6% 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

11 citing papers in PubMed, 26 citations in OpenAlex.

  1. Review
  2. Review
  3. Review
  4. Review
  5. Review
  6. Article
  7. Review
  8. Review
  9. Review
  10. Review
  11. 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

5 authors at 2 institutions in 2 countries.

Simone P CarneiroDepartment of Pharmacy, Pharmaceutical Technology and Biopharmaceutics, Ludwig-Maximilians-University of Munich, Munich, Germany.
Antonietta GrecoUniversity School for Advanced Studies (IUSS), Pavia, Italy.ORCID 0000-0001-7220-6911
Enrica ChiesaDepartment of Drug Sciences, University of Pavia, Pavia, Italy.
Ida GentaDepartment of Drug Sciences, University of Pavia, Pavia, Italy.ORCID 0000-0001-5710-0588
Olivia M MerkelDepartment of Pharmacy, Pharmaceutical Technology and Biopharmaceutics, Ludwig-Maximilians-University of Munich, Munich, Germany.ORCID 0000-0002-4151-3916
University of Pavia · ITLudwig-Maximilians-Universität München · DE

Funding

European Research Council 101069308European Research Council 637830
6 · The paper itself

Abstract

introductionMost lung diseases are serious conditions resulting from genetic and environmental causes associated with high mortality and severe symptoms. Currently, treatments available have a palliative effect and many targets are still considered undruggable. Gene therapy stands as an attractive approach to offering innovative therapeutic solutions. CRISPRCas9 has established a remarkable potential for genome editing with high selectivity to targeted mutations. To ensure high efficacy with minimum systemic exposure, the delivery and administration route are key components that must be investigated. AREAS COVERED: This review is focused on the delivery of CRISPRCas9 to the lungs, taking advantage of lipid nanoparticles (LNPs), the most clinically advanced nucleic acid carriers. We also aim to highlight the benefits of pulmonary administration as a local delivery route and the use of spray drying to prepare stable nucleic-acid-based dry powder formulations that can overcome multiple lung barriers. EXPERT OPINION: Exploring the pulmonary administration to deliver CRISPRCas9 loaded in LNPs as a dry powder increases the chances to achieve high efficacy and reduced adverse effects. CRISPRCas9 loaded in LNP-embedded microparticles has not yet been reported in the literature but has the potential to reach and accumulate in target cells in the lung, thus, enhancing overall efficacy and safety.

Indexed as

Lung DiseasesNanoparticlesAdministration, InhalationCRISPR-Cas SystemsDry Powder InhalersHumansLiposomesLungParticle SizePowdersLipid NanoparticlesLiposomesPowdersCRISPR-Cas9Gene therapylipid nanoparticlespulmonary administrationspray drying

Identifiers

PMID36896650
PMCPMC7614984
OpenAlexW4323804928

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.