Evidence map›Paper›PMID 39155863›Full record

ArticleCirculation2024

Antisense Oligonucleotide Therapy for Calmodulinopathy.

Raul H Bortolin, Farina Nawar, Chaehyoung Park, Michael A Trembley, Maksymilian Prondzynski, Mason E Sweat, Peizhe Wang, Jiehui Chen, Fujian Lu, Carter Liou and 20 more

Abstract read
In one paragraph

Article in Circulation, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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

12 citing papers in PubMed.

  1. Review
  2. Review
  3. Article
  4. Review
  5. CALM1, CALM2, and CALM3 expression and translation efficiency provide insight into the severity of calmodulinopathy.Europace : European pacing, arrhythmias, and cardiac electrophysiology : journal of the working groups on cardiac pacing, arrhythmias, and cardiac cellular electrophysiology of the European Society of Cardiology · 2026
    Article
  6. Review
  7. Gene therapy for cardiac arrhythmias.Nature reviews. Cardiology · 2026
    Review
  8. Article
  9. Gene Therapy for Inherited Cardiac Arrhythmias.Journal of cardiovascular translational research · 2025
    Review
  10. Review
  11. Review
  12. 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

30 authors.

Raul H BortolinDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Farina NawarDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Chaehyoung ParkDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0009-0006-2391-1300
Michael A TrembleyDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Maksymilian ProndzynskiDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Mason E SweatDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0002-5400-1266
Peizhe WangDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0003-4298-2471
Jiehui ChenDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0002-0550-2195
Fujian LuDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0003-3605-3924
Carter LiouDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0009-0000-2577-4350
Paul BerksonDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Erin M KeatingDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).
Daisuke YoshinagaDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0003-0484-510X
Nikoleta PavlakiDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0001-5484-192X
Thomas SamenukDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0001-9651-4433
Cecilia B CavazzoniBoston Children's Hospital, MA. Transplantation Research Center, Renal Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA (C.B.C., P.T.S.).ORCID 0000-0003-2113-6620
Peter T SageBoston Children's Hospital, MA. Transplantation Research Center, Renal Division, Brigham and Women's Hospital, Harvard Medical School, Boston, MA (C.B.C., P.T.S.).ORCID 0000-0002-7287-0326
Qing MaDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0002-5625-1330
Robert D WhitehillDepartment of Pediatrics, Division of Cardiology, Emory University School of Medicine, Children's Healthcare of Atlanta, GA (R.D.W.).ORCID 0000-0002-3810-7132
Dominic J AbramsDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0001-5266-2805
Chrystalle Katte CarreonCardiac Registry, Departments of Cardiology, Pathology, and Cardiac Surgery (C.K.C.).ORCID 0000-0003-2152-7998
Juan PutraDepartment of Pathology (C.K.C., J.P., S.A.), Boston Children's Hospital and Harvard Medical School, MA.
Sanda AlexandrescuDepartment of Pathology (C.K.C., J.P., S.A.), Boston Children's Hospital and Harvard Medical School, MA.ORCID 0000-0001-9246-4184
Shuai GuoWells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).ORCID 0000-0002-9368-0548
Wen-Chin TsaiWells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).
Michael RubartWells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).ORCID 0000-0002-7386-6943
Dieter A KubliIonis Pharmaceuticals, Carlsbad, CA (D.A.K., A.E.M.).ORCID 0000-0001-7970-4075
Adam E MullickIonis Pharmaceuticals, Carlsbad, CA (D.A.K., A.E.M.).ORCID 0000-0003-4600-961X
Vassilios J BezzeridesDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).ORCID 0000-0003-0825-6580
William T PuDepartment of Cardiology (R.H.B., F.N., C.P., M.A.T., M.P., M.E.S., P.W., J.C., F.L., C.L., P.B., E.M.K., D.Y., N.P., T.S., Q.M., D.J.A., V.J.B., W.T.P.).

Funding

RESEARCH METHODS IN PEDIATRICS HEART DISEASET32HL007572 · NHLBI · CHILDREN'S HOSPITAL BOSTON · PI Sarah D DE FERRANTI, Jane W. Newburger · 1985 to 2026
$13.0M
Tissue chips for precision treatment of catecholaminergic polymorphic ventricular tachycardia - Diversity Supplement for Nnaemeka Justin AnyanwuUH3TR003279 · NCATS · BOSTON CHILDREN'S HOSPITAL · PI PARKER, KEVIN KIT, PU, WILLIAM TSWENCHING · 2022 to 2024
$2.5M
NCATS NIH HHS UH3 TR003279NHLBI NIH HHS T32 HL007572
6 · The paper itself

Abstract

backgroundCalmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in

methodsWe tested this hypothesis using human induced pluripotent stem cell-derived cardiomyocyte and mouse models of

resultsHuman

conclusionsThese results provide proof of concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies.

Indexed as

CalmodulinMyocytes, CardiacOligonucleotides, AntisenseAction PotentialsAnimalsDisease Models, AnimalGenetic TherapyHumansInduced Pluripotent Stem CellsLong QT SyndromeMiceMice, KnockoutCALM1 protein, humanCalmodulinOligonucleotides, Antisenseantisense oligonucleotidecalciumlong QT syndromeprecision medicinetachycardia, ventricular

Identifiers

PMID39155863
PMCPMC11747850

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