Evidence map›Paper›PMID 40409263›Full record

ArticleCell reports. Medicine2025

Cross-species tropism of AAV.CPP.16 in the respiratory tract and its gene therapies against pulmonary fibrosis and viral infection.

Zhi Yang, Yizheng Yao, Xi Chen, Victoria Madigan, Shanrui Pu, Xianqun Fan, Jun Pu, Fengfeng Bei

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Cell reports. Medicine, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–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

2 citing papers in PubMed.

  1. Review
  2. 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

8 authors.

Zhi YangDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA; Department of Ophthalmology, Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai 200011, China.
Yizheng YaoDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA; Jiangsu Key Laboratory of Neuropsychiatric Diseases and Institute of Neuroscience, Soochow University, Clinical Research Center of Neurological Disease, The Second Affiliated Hospital of Soochow University, Suzhou 215123, China.
Xi ChenDepartment of Neurosurgery, The Second Affiliated Hospital of Kunming Medical University, Kunming 650106, China; NHC Key Laboratory of Drug Addiction Medicine, Kunming Medical University, Kunming 650500, China.
Victoria MadiganBroad Institute of MIT and Harvard, Cambridge, MA 02142, USA; Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Shanrui PuDepartment of Neurosurgery, The Second Affiliated Hospital of Kunming Medical University, Kunming 650106, China; NHC Key Laboratory of Drug Addiction Medicine, Kunming Medical University, Kunming 650500, China.
Xianqun FanDepartment of Ophthalmology, Ninth People's Hospital, Shanghai JiaoTong University School of Medicine, Shanghai 200011, China. Electronic address: fanxq@sjtu.edu.cn.
Jun PuDepartment of Neurosurgery, The Second Affiliated Hospital of Kunming Medical University, Kunming 650106, China; NHC Key Laboratory of Drug Addiction Medicine, Kunming Medical University, Kunming 650500, China. Electronic address: pujun137@126.com.
Fengfeng BeiDepartment of Neurosurgery, Brigham and Women's Hospital, Harvard Medical School, Boston, MA 02115, USA. Electronic address: fbei@bwh.harvard.edu.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Efficient gene delivery vectors are crucial for respiratory and lung disease therapies. We report that AAV.CPP.16, an engineered adeno-associated virus (AAV) variant derived from AAV9, efficiently transduces airway and lung cells in mice and non-human primates via intranasal administration. AAV.CPP.16 outperforms AAV6 and AAV9, two wild-type AAVs with demonstrated tropism for respiratory tissues, and efficiently targets key respiratory cell types. It supports gene supplementation and editing therapies in two clinically relevant mouse models of respiratory and lung diseases. A single intranasal dose of AAV.CPP.16 expressing a dual-target, vascular endothelial growth factor (VEGF)/transforming growth factor (TGF)-β1-neutralizing protein protected lungs from idiopathic pulmonary fibrosis, while a similar application of AAV.CPP.16 carrying an "all-in-one" CRISPR-Cas13d system inhibited transcription of the SARS-CoV-2-derived RNA-dependent RNA polymerase (Rdrp) gene. Our findings highlight AAV.CPP.16 as a promising vector for respiratory and lung gene therapy.

Indexed as

DependovirusGenetic TherapyIdiopathic Pulmonary FibrosisPulmonary FibrosisRespiratory SystemViral TropismVirus DiseasesAdministration, IntranasalAnimalsCOVID-19CRISPR-Cas SystemsDisease Models, AnimalFemaleGene EditingGenetic VectorsHumansVascular Endothelial Growth Factor AAAVCRISPRlung gene therapypulmonary fibrosis

Identifiers

PMID40409263
PMCPMC12208343

What OpenQuestion holds

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Registered trials

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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.