PMID- 29336355
OWN - NLM
STAT- PubMed-not-MEDLINE
LR  - 20240323
IS  - 1674-8301 (Print)
IS  - 2352-4685 (Electronic)
IS  - 1674-8301 (Linking)
VI  - 32
IP  - 2
DP  - 2018 Mar 26
TI  - Evaluation of cardiolipin nanodisks as lipid replacement therapy for Barth 
      syndrome.
PG  - 107-112
LID - 10.7555/JBR.32.20170094 [doi]
AB  - Barth syndrome (BTHS) is a mitochondrial disorder characterized by cardiomyopathy 
      and skeletal muscle weakness. Disease results from mutations in the tafazzin 
      (TAZ) gene, encoding a phospholipid transacylase. Defective tafazzin activity 
      results in an aberrant cardiolipin (CL) profile. The feasibility of restoring the 
      intracellular CL profile was tested by in vivo administration of exogenous CL in 
      nanodisk (ND) delivery particles. Ninety mg/kg CL (as ND) was administered to 
      doxycycline-inducible taz shRNA knockdown (KD) mice once a week. After 10 weeks 
      of CL-ND treatment, the mice were sacrificed and tissues harvested. Liquid 
      chromatography-mass spectrometry of extracted lipids revealed that CL-ND 
      administration failed to alter the CL profile of taz KD or WT mice. Thus, 
      although CL-ND were previously shown to be an effective means of delivering CL to 
      cultured cells, this effect does not extend to an in vivo setting. We conclude 
      that CL-ND administration is not a suitable therapy option for BTHS.
FAU - Ikon, Nikita
AU  - Ikon N
AD  - Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA.
FAU - Hsu, Fong-Fu
AU  - Hsu FF
AD  - Department of Medicine, School of Medicine, Washington University, St. Louis, MO 
      63110, USA.
FAU - Shearer, Jennifer
AU  - Shearer J
AD  - Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA.
FAU - Forte, Trudy M
AU  - Forte TM
AD  - Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA.
FAU - Ryan, Robert O
AU  - Ryan RO
AD  - Children's Hospital Oakland Research Institute, Oakland, CA 94609, USA.
AD  - Department of Biochemistry, University of Nevada, Reno, NV 89557, USA.
LA  - eng
GR  - R37 HL064159/HL/NHLBI NIH HHS/United States
GR  - R21 HL120760/HL/NHLBI NIH HHS/United States
GR  - P30 DK056341/DK/NIDDK NIH HHS/United States
GR  - P41 GM103422/GM/NIGMS NIH HHS/United States
GR  - P30 DK020579/DK/NIDDK NIH HHS/United States
PT  - Journal Article
PL  - China
TA  - J Biomed Res
JT  - Journal of biomedical research
JID - 101551157
PMC - PMC5895564
COIS- The authors reported no conflict of interests.
EDAT- 2018/01/18 06:00
MHDA- 2018/01/18 06:01
PMCR- 2018/03/26
CRDT- 2018/01/17 06:00
PHST- 2018/01/17 06:00 [entrez]
PHST- 2018/01/18 06:00 [pubmed]
PHST- 2018/01/18 06:01 [medline]
PHST- 2018/03/26 00:00 [pmc-release]
AID - 10.7555/JBR.32.20170094 [doi]
PST - ppublish
SO  - J Biomed Res. 2018 Mar 26;32(2):107-112. doi: 10.7555/JBR.32.20170094.