Evidence map›Paper›PMID 33752441›Full record

ArticleHuman gene therapy2021

Inmaculada de Melo-Martin, Ronald G Crystal

Open access · greenAbstract read
In one paragraph

Article in Human gene therapy, 2021. 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
0.2field-weighted citation impact, top 55% 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

2 citing papers in PubMed, 3 citations in OpenAlex.

  1. Article
  2. Article
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

2 authors at 1 institution in 1 country.

Inmaculada de Melo-MartinDivision of Medical Ethics, Department of Medicine, New York, New York, USA.ORCID 0000-0001-8656-8497
Ronald G CrystalDepartment of Genetic Medicine, Weill Cornell Medical College, New York, New York, USA.ORCID 0000-0001-6177-6839
Cornell University · US

Funding

Gene Therapy to Treat Ethanol-induced Osteoporosis Associated with Aldehyde Dehydrogenase 2 DeficiencyR41AA028465 · NIAAA · LEXEO THERAPEUTICS, LLC · PI CRYSTAL, RONALD G · 2020 to 2020
$252k
Gene Therapy to Prevent Esophageal Cancer Associated with Aldehyde Dehydrogenase DeficiencyR41AA027739 · NIAAA · LEXEO THERAPEUTICS, LLC · PI CRYSTAL, RONALD G · 2019 to 2019
$225k
NIAAA NIH HHS R41 AA027739NIAAA NIH HHS R41 AA028465
6 · The paper itself

Abstract

Aldehyde dehydrogenase 2 (ALDH2) deficiency constitutes one of the most common hereditary enzyme deficiencies, affecting 35% to 40% of East Asians and 8% of the world population. It causes the well-known Asian Alcohol Flush Syndrome, characterized by facial flushing, palpitation, tachycardia, nausea, and other unpleasant feelings when alcohol is consumed. It is also associated with a marked increase in the risk of a variety of serious disorders, including esophageal cancer and osteoporosis. Our recent studies with murine models have demonstrated that a one-time administration of an adeno-associated virus (AAV) gene transfer vector expressing the human ALDH2 coding sequence (AAVrh.10hALDH2) will correct the deficiency state and prevent alcohol-induced abnormalities of the esophagus and bone. If successful in humans, such strategy would reduce the increased risk-associated disorders such as esophageal cancer and osteoporosis, but also prevent the Asian Alcohol Flush Syndrome. This treatment thus raises ethical concerns: although it would potentially prevent fatal disease, it could also allow affected individuals to drink alcohol without suffering the Asian Alcohol Flush Syndrome and, hence, potentially enable personal destructive behavior. Here we explore the ethical arguments against the development of a gene therapy for ALDH2 deficiency and we find them wanting. We contend that development of such treatments is ethically appropriate and should be part and parcel of the solutions offered against the condition.

Indexed as

FlushingGenetic TherapyAldehyde Dehydrogenase, MitochondrialAnimalsAsian PeopleEsophagusEthanolHumansMiceAldehyde Dehydrogenase, MitochondrialALDH2 protein, humanEthanolALDH2 deficiencyAsian Alcohol Flush Syndromeethicsgene therapy

Identifiers

PMID33752441
PMCPMC8236557
OpenAlexW3136817056

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.