4.8 Article

Ionizable lipid nanoparticles for in utero mRNA delivery

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Cell Biology

In utero gene editing for monogenic lung disease

Deepthi Alapati et al.

SCIENCE TRANSLATIONAL MEDICINE (2019)

Article Pediatrics

Fetal intervention for congenital diaphragmatic hernia

Mark L. Kovler et al.

SEMINARS IN PEDIATRIC SURGERY (2019)

Article Multidisciplinary Sciences

Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

Brian Truong et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Chemistry, Multidisciplinary

Optimizing biodegradable nanoparticle size for tissue-specific delivery

Hanna K. Mandl et al.

JOURNAL OF CONTROLLED RELEASE (2019)

Article Obstetrics & Gynecology

Fetal anemia: Diagnosis and management

Federico Prefumo et al.

BEST PRACTICE & RESEARCH CLINICAL OBSTETRICS & GYNAECOLOGY (2019)

Article Pharmacology & Pharmacy

Microfluidics-Assisted Size Tuning and Biological Evaluation of PLGA Particles

Maria Camilla Operti et al.

PHARMACEUTICS (2019)

Review Biotechnology & Applied Microbiology

Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

Zeljka Trepotec et al.

MOLECULAR THERAPY (2019)

Article Chemistry, Multidisciplinary

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

Pedro P. G. Guimaraes et al.

JOURNAL OF CONTROLLED RELEASE (2019)

Review Biotechnology & Applied Microbiology

Delivery technologies for cancer immunotherapy

Rachel S. Riley et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Review Materials Science, Biomaterials

Biomaterials in fetal surgery

Sally M. Winkler et al.

BIOMATERIALS SCIENCE (2019)

Article Biochemical Research Methods

Tuning the Size of Poly(lactic-co-glycolic Acid) (PLGA) Nanoparticles Fabricated by Nanoprecipitation

Wei Huang et al.

BIOTECHNOLOGY JOURNAL (2018)

Review Chemistry, Multidisciplinary

Advances in Biomaterials for Drug Delivery

Owen S. Fenton et al.

ADVANCED MATERIALS (2018)

Article Biochemistry & Molecular Biology

Fetal gene therapy for neurodegenerative disease of infants

Giulia Massaro et al.

NATURE MEDICINE (2018)

Review Biotechnology & Applied Microbiology

mRNA vaccines - a new era in vaccinology

Norbert Pardi et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

Article Medicine, General & Internal

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

D. Adams et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Review Pharmacology & Pharmacy

Engineered nanomaterial applications in perinatal therapeutics

S. B. Fournier et al.

PHARMACOLOGICAL RESEARCH (2018)

Article Multidisciplinary Sciences

In utero nanoparticle delivery for site-specific genome editing

Adele S. Ricciardi et al.

NATURE COMMUNICATIONS (2018)

Review Biochemistry & Molecular Biology

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

Jayesh A. Kulkarni et al.

NUCLEIC ACID THERAPEUTICS (2018)

News Item Biotechnology & Applied Microbiology

Alnylam launches era of RNAi drugs

Ken Garber

NATURE BIOTECHNOLOGY (2018)

Editorial Material Multidisciplinary Sciences

The fix is in utero

Sarah DeWeerdt

NATURE (2018)

Article Biochemistry & Molecular Biology

In utero CRISPR-mediated therapeutic editing of metabolic genes

Avery C. Rossidis et al.

NATURE MEDICINE (2018)

Article Biochemistry & Molecular Biology

SERPINA1 mRNA as a Treatment for Alpha-1 Antitrypsin Deficiency

Brendan Connolly et al.

JOURNAL OF NUCLEIC ACIDS (2018)

Review Pharmacology & Pharmacy

Potential of Peptide Nucleic Acids in Future Therapeutic Applications

Soheila Montazersaheb et al.

ADVANCED PHARMACEUTICAL BULLETIN (2018)

Article Pharmacology & Pharmacy

Quantitative Systems Pharmacology Model of hUGT1A1-modRNA Encoding for the UGT1A1 Enzyme to Treat Crigler-Najjar Syndrome Type 1

Joshua F. Apgar et al.

CPT-PHARMACOMETRICS & SYSTEMS PHARMACOLOGY (2018)

Article Pharmacology & Pharmacy

Enzyme replacement therapies: what is the best option?

Azam Safary et al.

BIOIMPACTS (2018)

Article Chemistry, Multidisciplinary

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Matthias A. Oberli et al.

NANO LETTERS (2017)

Article Obstetrics & Gynecology

Committee Opinion No. 690

OBSTETRICS AND GYNECOLOGY (2017)

Article Multidisciplinary Sciences

Lipophilic siRNA targets albumin in situ and promotes bioavailability, tumor penetration, and carrier-free gene silencing

Samantha M. Sarett et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Article Multidisciplinary Sciences

Systemic delivery of factor IX messenger RNA for protein replacement therapy

Suvasini Ramaswamy et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Editorial Material Acoustics

Fetal anemia

N. Abbasi et al.

ULTRASOUND IN OBSTETRICS & GYNECOLOGY (2017)

Review Gastroenterology & Hepatology

Macrophage inflammatory protein-2 as mediator of inflammation in acute liver injury

Chao-Chao Qin et al.

WORLD JOURNAL OF GASTROENTEROLOGY (2017)

Review Nanoscience & Nanotechnology

Tools for translation: non-viral materials for therapeutic mRNA delivery

Khalid A. Hajj et al.

NATURE REVIEWS MATERIALS (2017)

Article Obstetrics & Gynecology

In utero stem cell transplantation and gene therapy: Recent progress and the potential for clinical application

Lauren E. McClain et al.

BEST PRACTICE & RESEARCH CLINICAL OBSTETRICS & GYNAECOLOGY (2016)

Article Biochemistry & Molecular Biology

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

F. DeRosa et al.

GENE THERAPY (2016)

Review Medicine, Research & Experimental

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

Graca Almeida-Porada et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)

Article Chemistry, Multidisciplinary

Dendrimer-Inspired Nanomaterials for the in Vivo Delivery of siRNA to Lung Vasculature

Omar F. Khan et al.

NANO LETTERS (2015)

Article Multidisciplinary Sciences

Degradable lipid nanoparticles with predictable in vivo siRNA delivery activity

Kathryn A. Whitehead et al.

NATURE COMMUNICATIONS (2014)

Review Neurosciences

Perinatal systemic gene delivery using adeno-associated viral vectors

Rajvinder Karda et al.

FRONTIERS IN MOLECULAR NEUROSCIENCE (2014)

Review Pharmacology & Pharmacy

Liposomal drug delivery systems: From concept to clinical applications

Theresa M. Allen et al.

ADVANCED DRUG DELIVERY REVIEWS (2013)

Editorial Material Cell Biology

Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

Jennifer A. Gorzelany et al.

SCIENCE TRANSLATIONAL MEDICINE (2013)

Article Chemistry, Multidisciplinary

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

Muthusamy Jayaraman et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Review Chemistry, Multidisciplinary

PLGA-based nanoparticles: An overview of biomedical applications

Fabienne Danhier et al.

JOURNAL OF CONTROLLED RELEASE (2012)

Article Chemistry, Multidisciplinary

Rapid Discovery of Potent siRNA-Containing Lipid Nanoparticles Enabled by Controlled Microfluidic Formulation

Delai Chen et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2012)

Article Medicine, General & Internal

A Randomized Trial of Prenatal versus Postnatal Repair of Myelomeningocele

N. Scott Adzick et al.

NEW ENGLAND JOURNAL OF MEDICINE (2011)

Article Multidisciplinary Sciences

Lipid-like materials for low-dose, in vivo gene silencing

Kevin T. Love et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)

Article Pediatrics

Stem cell and genetic therapies for the fetus

Jessica L. Roybal et al.

SEMINARS IN FETAL & NEONATAL MEDICINE (2010)

Article Medicine, Research & Experimental

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

Steven J. Howe et al.

JOURNAL OF CLINICAL INVESTIGATION (2008)

Article Medicine, Research & Experimental

Factors affecting the clearance and biodistribution of polymeric nanoparticles

Frank Alexis et al.

MOLECULAR PHARMACEUTICS (2008)

Article Chemistry, Multidisciplinary

Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

J Heyes et al.

JOURNAL OF CONTROLLED RELEASE (2005)

Article Critical Care Medicine

Innate immune response in Th1- and Th2-dominant mouse strains

H Watanabe et al.