标题
Highly branched poly(β-amino ester)s for gene delivery in hereditary skin diseases
作者
关键词
Cutaneous gene delivery, Polymeric vectors, Highly branched poly(β-amino ester)s, Hereditary skin diseases, Recessive dystrophic epidermolysis bullosa
出版物
ADVANCED DRUG DELIVERY REVIEWS
Volume 176, Issue -, Pages 113842
出版商
Elsevier BV
发表日期
2021-07-20
DOI
10.1016/j.addr.2021.113842
参考文献
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。- Sharing (data) is caring for patients with pachyonychia congenita
- (2020) D.A. Mordaunt BRITISH JOURNAL OF DERMATOLOGY
- What’s New in Genetic Skin Diseases
- (2019) Callie R. Hill et al. DERMATOLOGIC CLINICS
- Transcutaneous delivery of DNA/mRNA for cancer therapeutic vaccination
- (2019) Xiaoyi Sun et al. JOURNAL OF GENE MEDICINE
- Highly branched poly(β-amino ester) delivery of minicircle DNA for transfection of neurodegenerative disease related cells
- (2019) Shuai Liu et al. Nature Communications
- Efficient and Robust Highly Branched Poly(β-amino ester)/Minicircle COL7A1 Polymeric Nanoparticles for Gene Delivery to Recessive Dystrophic Epidermolysis Bullosa Keratinocytes
- (2019) Ming Zeng et al. ACS Applied Materials & Interfaces
- Poly(β-amino ester)-based gene delivery systems: From discovery to therapeutic applications
- (2019) Rosemeyre A. Cordeiro et al. JOURNAL OF CONTROLLED RELEASE
- EB2017—Progress in Epidermolysis Bullosa Research toward Treatment and Cure
- (2018) Jouni Uitto et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Epidermolysis bullosa: Molecular pathology of connective tissue components in the cutaneous basement membrane zone
- (2018) Cristina Has et al. MATRIX BIOLOGY
- Recent advances in ultrasound-based transdermal drug delivery
- (2018) Brenden Cheong-Qi Seah et al. International Journal of Nanomedicine
- Manipulation of Transgene Expression in Fibroblast Cells by a Multifunctional Linear-Branched Hybrid Poly(β-Amino Ester) Synthesized through an Oligomer Combination Approach
- (2018) Ming Zeng et al. NANO LETTERS
- Nanotechnologies in delivery of mRNA therapeutics using nonviral vector-based delivery systems
- (2017) S Guan et al. GENE THERAPY
- Gentamicin induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients
- (2017) David T. Woodley et al. JOURNAL OF CLINICAL INVESTIGATION
- Do nanoparticles have a future in dermal drug delivery?
- (2017) Alexa Patzelt et al. JOURNAL OF CONTROLLED RELEASE
- Topical and cutaneous delivery using nanosystems
- (2017) MS Roberts et al. JOURNAL OF CONTROLLED RELEASE
- Closure of a Large Chronic Wound through Transplantation of Gene-Corrected Epidermal Stem Cells
- (2017) Johann W. Bauer et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa
- (2017) Stefan Hainzl et al. MOLECULAR THERAPY
- Gene therapy: Transgenic stem cells replace skin
- (2017) Mariaceleste Aragona et al. NATURE
- Regeneration of the entire human epidermis using transgenic stem cells
- (2017) Tobias Hirsch et al. NATURE
- Antisense oligonucleotides: the next frontier for treatment of neurological disorders
- (2017) Carlo Rinaldi et al. Nature Reviews Neurology
- Highly Branched poly(5-amino-1-pentanol-co-1,4-butanediol diacrylate) for High Performance Gene Transfection
- (2017) Ming Zeng et al. Polymers
- Development of Branched Poly(5-Amino-1-pentanol-co-1,4-butanediol Diacrylate) with High Gene Transfection Potency Across Diverse Cell Types
- (2016) Dezhong Zhou et al. ACS Applied Materials & Interfaces
- PEGylation as a strategy for improving nanoparticle-based drug and gene delivery
- (2016) Jung Soo Suk et al. ADVANCED DRUG DELIVERY REVIEWS
- Highly Branched Poly(β-amino esters) for Non-Viral Gene Delivery: High Transfection Efficiency and Low Toxicity Achieved by Increasing Molecular Weight
- (2016) Yongsheng Gao et al. BIOMACROMOLECULES
- A non-viral gene therapy for treatment of recessive dystrophic epidermolysis bullosa
- (2016) Lara Cutlar et al. EXPERIMENTAL DERMATOLOGY
- Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa
- (2016) Zurab Siprashvili et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Highly branched poly(β-amino ester)s for skin gene therapy
- (2016) Dezhong Zhou et al. JOURNAL OF CONTROLLED RELEASE
- Non-viral gene therapy: Gains and challenges of non-invasive administration methods
- (2016) Marianna Foldvari et al. JOURNAL OF CONTROLLED RELEASE
- Targeted Exon Skipping Restores Type VII Collagen Expression and Anchoring Fibril Formation in an In Vivo RDEB Model
- (2016) Sandrina Turczynski et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Lentiviral Engineered Fibroblasts Expressing Codon-Optimized COL7A1 Restore Anchoring Fibrils in RDEB
- (2016) Christos Georgiadis et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Genotypic Heterogeneity and the Mode of Inheritance in Epidermolysis Bullosa
- (2016) Jouni Uitto et al. JAMA Dermatology
- Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes
- (2016) Cristina Chamorro et al. Molecular Therapy-Nucleic Acids
- The transition from linear to highly branched poly( -amino ester)s: Branching matters for gene delivery
- (2016) D. Zhou et al. Science Advances
- Minicircle DNA Provides Enhanced and Prolonged Transgene Expression Following Airway Gene Transfer
- (2016) Mustafa M. Munye et al. Scientific Reports
- Polymeric Nanoparticles for Nonviral Gene Therapy Extend Brain Tumor Survival in Vivo
- (2015) Antonella Mangraviti et al. ACS Nano
- Highly Branched Poly(β-Amino Esters): Synthesis and Application in Gene Delivery
- (2015) Lara Cutlar et al. BIOMACROMOLECULES
- Tailoring highly branched poly(β-amino ester)s: a synthetic platform for epidermal gene therapy
- (2015) Jian-Yuan Huang et al. CHEMICAL COMMUNICATIONS
- Losartan ameliorates dystrophic epidermolysis bullosa and uncovers new disease mechanisms
- (2015) A. Nystrom et al. EMBO Molecular Medicine
- Recessive Dystrophic Epidermolysis Bullosa: Advances in the Laboratory Leading to New Therapies
- (2015) David T. Woodley et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa
- (2015) Gabriela Petrof et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- High Local Concentrations of Intradermal MSCs Restore Skin Integrity and Facilitate Wound Healing in Dystrophic Epidermolysis Bullosa
- (2015) Tobias Kühl et al. MOLECULAR THERAPY
- Enabling cytoplasmic delivery and organelle targeting by surface modification of nanocarriers
- (2015) Alessandro Parodi et al. Nanomedicine
- Gene therapy returns to centre stage
- (2015) Luigi Naldini NATURE
- A Biologic Velcro Patch
- (2015) Jakub Tolar et al. NEW ENGLAND JOURNAL OF MEDICINE
- Recent insights into cutaneous immunization: How to vaccinate via the skin
- (2015) Laura Engelke et al. VACCINE
- Biodegradable Polymeric Nanoparticles Show High Efficacy and Specificity at DNA Delivery to Human Glioblastoma in Vitro and in Vivo
- (2014) Hugo Guerrero-Cázares et al. ACS Nano
- Significance of Branching for Transfection: Synthesis of Highly Branched Degradable Functional Poly(dimethylaminoethyl methacrylate) by Vinyl Oligomer Combination
- (2014) Tianyu Zhao et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- Minicircle DNA vectors for gene therapy: advances and applications
- (2014) Vítor Gaspar et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- Therapeutic Potential of Human Adipose-Derived Stem Cells in Neurological Disorders
- (2014) Keun-A Chang et al. JOURNAL OF PHARMACOLOGICAL SCIENCES
- Inherited epidermolysis bullosa: Updated recommendations on diagnosis and classification
- (2014) Jo-David Fine et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Non-viral vectors for gene-based therapy
- (2014) Hao Yin et al. NATURE REVIEWS GENETICS
- Genetically corrected iPSCs as cell therapy for recessive dystrophic epidermolysis bullosa
- (2014) D. Wenzel et al. Science Translational Medicine
- A New Strategy to Deliver Synthetic Protein Drugs: Self-reproducible Biologics Using Minicircles
- (2014) Hyoju Yi et al. Scientific Reports
- Gene Therapy for Skin Diseases
- (2014) E. Gorell et al. Cold Spring Harbor Perspectives in Medicine
- Safety and efficacy of DNA vaccines
- (2014) Sofia Stenler et al. Human Vaccines & Immunotherapeutics
- Effect of Polyplex Morphology on Cellular Uptake, Intracellular Trafficking, and Transgene Expression
- (2013) Julie Shi et al. ACS Nano
- Degradable Terpolymers with Alkyl Side Chains Demonstrate Enhanced Gene Delivery Potency and Nanoparticle Stability
- (2013) Ahmed A. Eltoukhy et al. ADVANCED MATERIALS
- Fibroblast cell therapy enhances initial healing in recessive dystrophic epidermolysis bullosa wounds: results of a randomized, vehicle-controlled trial
- (2013) G. Petrof et al. BRITISH JOURNAL OF DERMATOLOGY
- Polymer gene delivery: overcoming the obstacles
- (2013) Ahmed Aied et al. DRUG DISCOVERY TODAY
- Microneedles for intradermal and transdermal drug delivery
- (2013) Tuan-Mazlelaa Tuan-Mahmood et al. EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
- Keratinocyte cell lines derived from severe generalized recessive Epidermolysis Bullosa patients carrying a highly recurrentCOL7A1homozygous mutation: models to assess cell and gene therapiesin vitroandin vivo
- (2013) Cristina Chamorro et al. EXPERIMENTAL DERMATOLOGY
- DNA delivery with hyperbranched polylysine: A comparative study with linear and dendritic polylysine
- (2013) Zuzana Kadlecova et al. JOURNAL OF CONTROLLED RELEASE
- Intravenously Injected Recombinant Human Type VII Collagen Homes to Skin Wounds and Restores Skin Integrity of Dystrophic Epidermolysis Bullosa
- (2013) David T. Woodley et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- A phase II randomized vehicle-controlled trial of intradermal allogeneic fibroblasts for recessive dystrophic epidermolysis bullosa
- (2013) Supriya S. Venugopal et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Topical Application of Recombinant Type VII Collagen Incorporates Into the Dermal–Epidermal Junction and Promotes Wound Closure
- (2013) Xinyi Wang et al. MOLECULAR THERAPY
- Long-Term Stability and Safety of Transgenic Cultured Epidermal Stem Cells in Gene Therapy of Junctional Epidermolysis Bullosa
- (2013) Laura De Rosa et al. Stem Cell Reports
- The effect of molecular weight, compositions and lectin type on the properties of hyperbranched glycopolymers as non-viral gene delivery systems
- (2012) Marya Ahmed et al. BIOMATERIALS
- Effect of molecular weight of amine end-modified poly(β-amino ester)s on gene delivery efficiency and toxicity
- (2012) Ahmed A. Eltoukhy et al. BIOMATERIALS
- Effective healing of diabetic skin wounds by using nonviral gene therapy based on minicircle vascular endothelial growth factor DNA and a cationic dendrimer
- (2012) Min J. Kwon et al. JOURNAL OF GENE MEDICINE
- Uptake and Transfection with Polymeric Nanoparticles Are Dependent on Polymer End-Group Structure, but Largely Independent of Nanoparticle Physical and Chemical Properties
- (2012) Joel C. Sunshine et al. MOLECULAR PHARMACEUTICS
- Non-viral gene delivery nanoparticles based on Poly(β-amino esters) for treatment of glioblastoma
- (2011) Stephany Y. Tzeng et al. BIOMATERIALS
- State-of-the-art gene-based therapies: the road ahead
- (2011) Mark A. Kay NATURE REVIEWS GENETICS
- A Novel Assay for Quantifying the Number of Plasmids Encapsulated by Polymer Nanoparticles
- (2011) Nupura S. Bhise et al. Small
- The relationship between terminal functionalization and molecular weight of a gene delivery polymer and transfection efficacy in mammary epithelial 2-D cultures and 3-D organotypic cultures
- (2010) Nupura S. Bhise et al. BIOMATERIALS
- Replenishment of type VII collagen and re-epithelialization of chronically ulcerated skin after intradermal administration of allogeneic mesenchymal stromal cells in two patients with recessive dystrophic epidermolysis bullosa
- (2010) Paulette Conget et al. CYTOTHERAPY
- Progress in Epidermolysis Bullosa Research: Toward Treatment and Cure
- (2010) Jouni Uitto et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Bone Marrow Transplantation for Recessive Dystrophic Epidermolysis Bullosa
- (2010) John E. Wagner et al. NEW ENGLAND JOURNAL OF MEDICINE
- Biosafety of DNA vaccines: New generation of DNA vectors and current knowledge on the fate of plasmids after injection
- (2010) Florence Faurez et al. VACCINE
- Nanoparticle-Delivered Suicide Gene Therapy Effectively Reduces Ovarian Tumor Burden in Mice
- (2009) Y.-H. Huang et al. CANCER RESEARCH
- Hydration Effects on Skin Microstructure as Probed by High-Resolution Cryo-Scanning Electron Microscopy and Mechanistic Implications to Enhanced Transcutaneous Delivery of Biomacromolecules
- (2009) Grace Tan et al. JOURNAL OF PHARMACEUTICAL SCIENCES
- Life with epidermolysis bullosa (EB): Etiology, diagnosis, multidisciplinary care and therapy
- (2009) Dedee F. Murrell JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Extracutaneous manifestations and complications of inherited epidermolysis bullosa
- (2009) Jo-David Fine et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Electroporation for the Delivery of DNA-based Vaccines and Immunotherapeutics: Current Clinical Developments
- (2009) Angela M Bodles-Brakhop et al. MOLECULAR THERAPY
- First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder
- (2009) Sancy A Leachman et al. MOLECULAR THERAPY
- Genetic engineering of human stem cells for enhanced angiogenesis using biodegradable polymeric nanoparticles
- (2009) Fan Yang et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- A hypomorphic mouse model of dystrophic epidermolysis bullosa reveals mechanisms of disease and response to fibroblast therapy
- (2008) Anja Fritsch et al. JOURNAL OF CLINICAL INVESTIGATION
- Epidermolysis bullosa and the risk of life-threatening cancers: The National EB Registry experience, 1986-2006
- (2008) Jo-David Fine et al. JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY
- Can Type VII Collagen Injections Cure Dystrophic Epidermolysis Bullosa?
- (2008) Leena Bruckner-Tuderman MOLECULAR THERAPY
- Injection of Recombinant Human Type VII Collagen Corrects the Disease Phenotype in a Murine Model of Dystrophic Epidermolysis Bullosa
- (2008) Jennifer Remington et al. MOLECULAR THERAPY
- Clearance properties of nano-sized particles and molecules as imaging agents: considerations and caveats
- (2008) Michelle Longmire et al. Nanomedicine
- Surface-structure-regulated cell-membrane penetration by monolayer-protected nanoparticles
- (2008) Ayush Verma et al. NATURE MATERIALS
- Sonoporation of the Minicircle-VEGF165 for Wound Healing of Diabetic Mice
- (2008) C. S. Yoon et al. PHARMACEUTICAL RESEARCH
- Efficient expression of vascular endothelial growth factor using minicircle DNA for angiogenic gene therapy
- (2007) Chien-Wen Chang et al. JOURNAL OF CONTROLLED RELEASE
Publish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn MoreCreate your own webinar
Interested in hosting your own webinar? Check the schedule and propose your idea to the Peeref Content Team.
Create Now