Evidence map›Paper›PMID 37394797›Full record

ArticleMolecular therapy : the journal of the American Society of Gene Therapy2023

Increased expression or activation of TRPML1 reduces hepatic storage of toxic Z alpha-1 antitrypsin.

Nunzia Pastore, Francesco Annunziata, Rita Colonna, Veronica Maffia, Teresa Giuliano, Bruno Maria Custode, Bernadette Lombardi, Elena Polishchuk, Vincenzo Cacace, Lucia De Stefano and 4 more

Open access · hybridAbstract read
In one paragraph

Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
1.3field-weighted citation impact, top 21% 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

6 citing papers in PubMed, 5 citations in OpenAlex.

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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

14 authors at 2 institutions in 1 country.

Nunzia PastoreTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy; Department of Translational Medicine, Medical Genetics, University of Naples Federico II, Naples, Italy. Electronic address: pastore@tigem.it.
Francesco AnnunziataTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Rita ColonnaTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Veronica MaffiaTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Teresa GiulianoTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Bruno Maria CustodeTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Bernadette LombardiTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Elena PolishchukTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Vincenzo CacaceTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Lucia De StefanoTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Edoardo NuscoTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Nicolina Cristina SorrentinoTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy; Department of Clinical Medicine and Surgery, University of Naples Federico II, Naples, Italy.
Pasquale PiccoloTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy.
Nicola Brunetti-PierriTelethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Naples, Italy; Department of Translational Medicine, Medical Genetics, University of Naples Federico II, Naples, Italy; Scuola Superiore Meridionale (SSM, School of Advanced Studies), Genomics and Experimental Medicine Program, University of Naples Federico II, Naples, Italy. Electronic address: brunetti@tigem.it.
Telethon Institute Of Genetics And Medicine · ITScuola Superiore Meridionale · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Mutant Z alpha-1 antitrypsin (ATZ) accumulates in globules in the liver and is the prototype of proteotoxic hepatic disease. Therapeutic strategies aiming at clearance of polymeric ATZ are needed. Transient receptor potential mucolipin-1 (TRPML1) is a lysosomal Ca

Indexed as

alpha 1-AntitrypsinLiver DiseasesTransient Receptor Potential ChannelsAnimalsHumansLysosomesMiceMice, Transgenicalpha 1-AntitrypsinMCOLN1 protein, humanMcoln1 protein, mouseTransient Receptor Potential Channelsalpha-1 antitrypsinalpha-1 antitrypsin deficiencylysosomal exocytosismucolipin-1TRPML1

Identifiers

PMID37394797
PMCPMC10492024
OpenAlexW4382810414

What OpenQuestion holds

Textmetadata
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.