Evidence map›Paper›PMID 35585789›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2022

Gene-based therapeutics for rare genetic neurodevelopmental psychiatric disorders.

Beverly L Davidson, Guangping Gao, Elizabeth Berry-Kravis, Allison M Bradbury, Carsten Bönnemann, Joseph D Buxbaum, Gavin R Corcoran, Steven J Gray, Heather Gray-Edwards, Robin J Kleiman and 8 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing 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

19 citing papers in PubMed.

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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

18 authors.

Beverly L DavidsonRaymond G. Perelman Center for Cellular and Molecular Therapeutics, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA, USA. Electronic address: davidsonbl@chop.edu.
Guangping GaoHorae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Li Weibo Institute for Rare Diseases Research, UMass Chan Medical School, Worcester, MA, USA; Department of Microbiology and Physiological Systems, UMass Chan Medical School, Worcester, MA, USA. Electronic address: guangping.gao@umassmed.edu.
Elizabeth Berry-KravisDepartments of Pediatrics, Neurological Sciences, Anatomy and Cell Biology, Rush University Medical Center, Chicago, IL, USA.
Allison M BradburyDepartment of Pediatrics, Abigail Wexner Research Institute Nationwide Children's Hospital, The Ohio State University, Columbus, OH, USA.
Carsten BönnemannNational Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.
Joseph D BuxbaumSeaver Autism Center for Research and Treatment, Department of Psychiatry, Icahn School of Medicine at Mount Sinai, New York, NY, USA.
Gavin R CorcoranTrialSpark, New York, NY, USA.
Steven J GrayDepartment of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA.
Heather Gray-EdwardsHorae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; Department of Radiology, UMass Chan Medical School, Worcester, MA, USA.
Robin J KleimanBiogen Research and Development, Cambridge, MA, USA.
Adam J ShaywitzBridgeBio Gene Therapy, Palo Alto, CA, USA.
Dan WangHorae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA; RNA Therapeutics Institute, UMass Chan Medical School, Worcester, MA, USA.
Huda Y ZoghbiHoward Hughes Medical Institute, Jan and Dan Duncan Neurological Research Institute, Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
Terence R FlotteHorae Gene Therapy Center, UMass Chan Medical School, Worcester, MA, USA.
Sitra Tauscher-WisniewskiNovartis Gene Therapies, Bannockburn, IL, USA.
Cynthia J TifftMedical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Mustafa SahinRosamund Stone Zander Translational Neuroscience Center, Department of Neurology, Boston Children's Hospital and Harvard Medical School, Boston, MA, USA. Electronic address: mustafa.sahin@childrens.harvard.edu.
Gene Therapy Workshop Faculty

Funding

Genetic Analysis and Manipulation Core (GAEC)P50HD105351 · NICHD · BOSTON CHILDREN'S HOSPITAL · PI SCOTT Loren POMEROY, MUSTAFA SAHIN · 2021 to 2026
$9.4M
Oligodendrocyte-focused rAAV gene therapy strategies for Canavan disease and LeukodystrophiesR01NS076991 · NINDS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI Guangping Gao · 2012 to 2026
$5.7M
Howard Hughes Medical InstituteNICHD NIH HHS P50 HD105351NINDS NIH HHS R01 NS076991
6 · The paper itself

Abstract

We are in an emerging era of gene-based therapeutics with significant promise for rare genetic disorders. The potential is particularly significant for genetic central nervous system disorders that have begun to achieve Food and Drug Administration approval for select patient populations. This review summarizes the discussions and presentations of the National Institute of Mental Health-sponsored workshop "Gene-Based Therapeutics for Rare Genetic Neurodevelopmental Psychiatric Disorders," which was held in January 2021. Here, we distill the points raised regarding various precision medicine approaches related to neurodevelopmental and psychiatric disorders that may be amenable to gene-based therapies.

Indexed as

Mental DisordersPrecision MedicineHumansRare DiseasesUnited StatesUnited States Food and Drug Administrationgene therapyneurodevelopmentpsychiatric disorders

Identifiers

PMID35585789
PMCPMC9263284

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.