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Microbubble Gene Therapy May Shield Against Heart Disease

Microbubble Gene Therapy May Shield Against Heart Disease
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Cynthia andersonStanford universityJohna burns school of medicineUniversity of hawaiCardiovascular researchMolecular therapy nucleic acidsRalph shohet

Using microbubble gene therapy may protect against heart disease | University of Hawaiʻi System News

Microbubbles were used to deliver CRISPR tools to the liver to inhibit a gene that increases cardiovascular risk.

Cynthia andersonUniversity of hawaiJohna burns school of medicineStanford universityCardiovascular researchMolecular therapy nucleic acidsRalph shohetUh manoaCenter for cardiovascular researchGene therapyCardiovascular diseaseManoa researchPublic impact research

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis

SiSaf Ltd, an RNA therapeutics company, is pleased to announce the publication in the peer-reviewed journal Molecular Therapy Nucleic Acids(1) of positive in vivo data demonstrating the safety and efficacy of its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The data reported in the paper demonstrate .

United statesAnna maria tetiSuzanne saffie siebertAntonio mauriziSisaf ltdMineral researchAmerican society of boneCell press journal molecular therapy nucleic acidsCell press journal molecular therapy nucleicMolecular therapy nucleic acidsAutosomal dominant osteopetrosisAmerican societyProfessor anna mariaProfessor tetiOrphan drug designationPediatric disease designation

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis

Peer-Reviewed Publication of Positive Preclinical Data of SiSaf s SIS-ADO2 siRNA Program to Treat Rare Genetic Bone Disorder Osteopetrosis
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United kingdomUnited statesSue charlesAnna maria tetiAntonio mauriziSuzanne saffie siebertMineral researchAmerican society of boneSisaf ltdReviewed publicationPositive preclinical dataTreat rare genetic bone disorderSaf ltdMolecular therapy nucleicAutosomal dominant osteopetrosisAmerican society

DNA-based drug to prevent blood clots

Researchers have proposed a side effect-free anticoagulating treatment that has so far proved effective in test mice and could be ready for human trials in just a few years.

Keitaro yoshimotoAsuka sakataUniversity of tokyoNara medical universityAssociate professor keitaro yoshimotoAssistant professor asuka sakataMolecular therapy nucleicBlood clotsHeparin induced thrombocytopeniaUniversity of tokyoMolecular therapy nucleic acidsMrna molecules