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Future Impact of mRNA Therapy on Cardiovascular Diseases [PDF]

Methodist DeBakey Cardiovascular Journal, 2022
The silver lining of the recent pandemic was that it accelerated the emergence of messenger ribonucleic acid (mRNA) therapeutics. The great promise of mRNA therapeutics was highlighted by the speed at which the vaccines were created, tested, and proven ...
John P. Cooke, Keith A. Youker
doaj   +5 more sources

mRNA therapy for myocardial infarction: A review of targets and delivery vehicles [PDF]

Frontiers in Bioengineering and Biotechnology, 2022
Cardiovascular diseases are the leading cause of death in the world. This is partly due to the low regenerative capacity of adult hearts. mRNA therapy is a promising approach under development for cardiac diseases.
Xinming Wang, Douglas H. Wu, Samuel E. Senyo   +2 more
doaj   +3 more sources

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemiaResearch in context [PDF]

EBioMedicine, 2019
Background: Isolated methylmalonic acidemia/aciduria (MMA) is an ultra-rare, serious, inherited metabolic disorder with significant morbidity and mortality.
Ding An, Andrea Frassetto, Eric Jacquinet, Marianne Eybye, Joseph Milano, Christine DeAntonis, Vi Nguyen, Rodrigo Laureano, Jaclyn Milton, Staci Sabnis, Christine M. Lukacs, Lin T. Guey   +11 more
doaj   +3 more sources

mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. [PDF]

Nat Commun, 2021
Glycogen Storage Disease 1a (GSD1a) is a rare, inherited metabolic disorder caused by deficiency of glucose 6-phosphatase (G6Pase-α). G6Pase-α is critical for maintaining interprandial euglycemia.
Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH.   +39 more
europepmc   +2 more sources

Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia. [PDF]

Nat Commun, 2020
Propionic acidemia/aciduria (PA) is an ultra-rare, life-threatening, inherited metabolic disorder caused by deficiency of the mitochondrial enzyme, propionyl-CoA carboxylase (PCC) composed of six alpha (PCCA) and six beta (PCCB) subunits.
Jiang L, Park JS, Yin L, Laureano R, Jacquinet E, Yang J, Liang S, Frassetto A, Zhuo J, Yan X, Zhu X, Fortucci S, Hoar K, Mihai C, Tunkey C, Presnyak V, Benenato KE, Lukacs CM, Martini PGV, Guey LT.   +19 more
europepmc   +2 more sources

Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4^-/- mouse model of PFIC3. [PDF]

J Hepatol, 2021
Background & Aims: Progressive familial intrahepatic cholestasis type 3 (PFIC3) is a rare lethal autosomal recessive liver disorder caused by loss-of-function variations of the ABCB4 gene, encoding a phosphatidylcholine transporter (ABCB4/MDR3 ...
Wei G, Cao J, Huang P, An P, Badlani D, Vaid KA, Zhao S, Wang DQ, Zhuo J, Yin L, Frassetto A, Markel A, Presnyak V, Gandham S, Hua S, Lukacs C, Finn PF, Giangrande PH, Martini PGV, Popov YV.   +19 more
europepmc   +2 more sources

Inhaled DNAI1 mRNA therapy for treatment of primary ciliary dyskinesia. [PDF]

Proc Natl Acad Sci U S A
Significance Primary ciliary dyskinesia (PCD) is caused by loss of ciliary activity and mucociliary clearance, resulting in recurrent respiratory tract infections and bronchiectasis, with no disease-modifying therapies available.
Hennig M, Bhattacharjee RB, Agarwal I, Alfaifi A, Casillas JE, Chavez S, Ishimaru D, Liston D, Mohapatra S, Molla T, Pathare S, Sidhu MS, Wang P, Wang Z, Lombana TN, Kharitonov VG, Couch JA, Lockhart DJ, Wustman BA.   +18 more
europepmc   +2 more sources

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria. [PDF]

Sci Transl Med
The urea cycle enzyme argininosuccinate lyase (ASL) enables the clearance of neurotoxic ammonia and the biosynthesis of arginine. Patients with ASL deficiency present with argininosuccinic aciduria, an inherited metabolic disease with hyperammonemia and ...
Gurung S, Timmermand OV, Perocheau D, Gil-Martinez AL, Minnion M, Touramanidou L, Fang S, Messina M, Khalil Y, Spiewak J, Barber AR, Edwards RS, Pinto PL, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Ridout D, Heywood W, Hargreaves I, Heales S, Mills PB, Waddington SN, Gissen P, Eaton S, Ryten M, Feelisch M, Frassetto A, Witney TH, Baruteau J.   +30 more
europepmc   +2 more sources

Single-dose mRNA therapy via biomaterial-mediated sequestration of overexpressed proteins. [PDF]

Sci Adv, 2020
Nanostructured mineral coatings delivering chemically modified or unmodified therapeutic mRNA improve diabetic wound healing. Nonviral mRNA delivery is an attractive therapeutic gene delivery strategy, as it achieves efficient protein overexpression in ...
Khalil AS, Yu X, Umhoefer JM, Chamberlain CS, Wildenauer LA, Diarra GM, Hacker TA, Murphy WL.   +7 more
europepmc   +2 more sources

Nonviral Delivery Systems of mRNA Vaccines for Cancer Gene Therapy

Pharmaceutics, 2022
In recent years, the use of messenger RNA (mRNA) in the fields of gene therapy, immunotherapy, and stem cell biomedicine has received extensive attention.
Yusi Wang, Rui Zhang, Lin Tang, Li Yang
doaj   +2 more sources