Antisense Oligonucleotide Therapy for Calmodulinopathy.

antisense oligonucleotide calmodulinopathy precision medicine

Journal

Circulation
ISSN: 1524-4539
Titre abrégé: Circulation
Pays: United States
ID NLM: 0147763

Informations de publication

Date de publication:
19 Aug 2024
Historique:
medline: 19 8 2024
pubmed: 19 8 2024
entrez: 19 8 2024
Statut: aheadofprint

Résumé

Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in We tested this hypothesis using human induced pluripotent stem cell-derived cardiomyocyte and mouse models of Human These results provide proof of concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies.

Sections du résumé

BACKGROUND UNASSIGNED
Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in
METHODS UNASSIGNED
We tested this hypothesis using human induced pluripotent stem cell-derived cardiomyocyte and mouse models of
RESULTS UNASSIGNED
Human
CONCLUSIONS UNASSIGNED
These results provide proof of concept that ASOs targeting individual calmodulin genes are potentially effective and safe therapies for calmodulinopathies.

Identifiants

DOI: 10.1161/CIRCULATIONAHA.123.068111 PMID: 39155863
pubmed: 39155863
doi: 10.1161/CIRCULATIONAHA.123.068111
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Auteurs

Raul H Bortolin (RH)

Department of Cardiology, Boston Children's Hospital, MA. (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 Nawar (F)

Department of Cardiology, Boston Children's Hospital, MA. (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 Park (C)

Department of Cardiology, Boston Children's Hospital, MA. (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 Trembley (MA)

Department of Cardiology, Boston Children's Hospital, MA. (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 Prondzynski (M)

Department of Cardiology, Boston Children's Hospital, MA. (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 Sweat (ME)

Department of Cardiology, Boston Children's Hospital, MA. (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 Wang (P)

Department of Cardiology, Boston Children's Hospital, MA. (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 Chen (J)

Department of Cardiology, Boston Children's Hospital, MA. (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 Lu (F)

Department of Cardiology, Boston Children's Hospital, MA. (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 Liou (C)

Department of Cardiology, Boston Children's Hospital, MA. (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 Berkson (P)

Department of Cardiology, Boston Children's Hospital, MA. (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 Keating (EM)

Department of Cardiology, Boston Children's Hospital, MA. (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 Yoshinaga (D)

Department of Cardiology, Boston Children's Hospital, MA. (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 Pavlaki (N)

Department of Cardiology, Boston Children's Hospital, MA. (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 Samenuk (T)

Department of Cardiology, Boston Children's Hospital, MA. (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 Cavazzoni (CB)

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 Sage (PT)

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 Ma (Q)

Department of Cardiology, Boston Children's Hospital, MA. (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 Whitehill (RD)

Department 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 Abrams (DJ)

Department of Cardiology, Boston Children's Hospital, MA. (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.).
Center for Cardiovascular Genetics, Boston Children's Hospital and Harvard Medical School, MA. (D.J.A., V.J.B., W.T.P.).
ORCID: 0000-0001-5266-2805

Chrystalle K Carreon (CK)

Cardiac Registry, Departments of Cardiology, Pathology, and Cardiac Surgery, Boston Children's Hospital, MA. (C.K.C.).
ORCID: 0000-0003-2152-7998

Juan Putra (J)

Department of Pathology, Boston Children's Hospital and Harvard Medical School, MA. (J.P., S.A.).

Sanda Alexandrescu (S)

Department of Pathology, Boston Children's Hospital and Harvard Medical School, MA. (J.P., S.A.).
ORCID: 0000-0001-9246-4184

Shuai Guo (S)

Wells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).
ORCID: 0000-0002-9368-0548

Wen-Chin Tsai (WC)

Wells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).

Michael Rubart (M)

Wells Centre for Pediatric Research, Indiana University School of Medicine, Indianapolis (S.G., W.-C.T., M.R.).
ORCID: 0000-0002-7386-6943

Dieter Kubli (D)

Ionis Pharmaceuticals, Carlsbad, CA (D.K., A.E.M.).
ORCID: 0000-0001-7970-4075

Adam E Mullick (AE)

Ionis Pharmaceuticals, Carlsbad, CA (D.K., A.E.M.).
ORCID: 0000-0003-4600-961X

Vassilios J Bezzerides (VJ)

Department of Cardiology, Boston Children's Hospital, MA. (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.).
Center for Cardiovascular Genetics, Boston Children's Hospital and Harvard Medical School, MA. (D.J.A., V.J.B., W.T.P.).
ORCID: 0000-0003-0825-6580

William T Pu (WT)

Department of Cardiology, Boston Children's Hospital, MA. (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.).
Center for Cardiovascular Genetics, Boston Children's Hospital and Harvard Medical School, MA. (D.J.A., V.J.B., W.T.P.).
Harvard Stem Cell Institute, Cambridge, MA (W.T.P.).

Classifications MeSH