Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system
出版年份 2022 全文链接
标题
Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system
作者
关键词
-
出版物
JOURNAL OF GENE MEDICINE
Volume -, Issue -, Pages -
出版商
Wiley
发表日期
2022-01-15
DOI
10.1002/jgm.3410
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- Progressive eye pathology in mucopolysaccharidosis type I mice and effects of enzyme replacement therapy
- (2020) Esteban A. Gonzalez et al. CLINICAL AND EXPERIMENTAL OPHTHALMOLOGY
- Haematopoietic stem cell gene therapy with IL ‐1Ra rescues cognitive loss in mucopolysaccharidosis IIIA
- (2020) Helen Parker et al. EMBO Molecular Medicine
- Genome Editing for Mucopolysaccharidoses
- (2020) Edina Poletto et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- The potential of gene therapy for mucopolysaccharidosis type I
- (2020) Luisa Natalia Pimentel Vera et al. Expert Opinion on Orphan Drugs
- Mucopolysaccharidosis Type I
- (2020) Francyne Kubaski et al. Diagnostics
- Nanoemulsions for “Nose-to-Brain” Drug Delivery
- (2019) Maria Bonferoni et al. Pharmaceutics
- Delivery Aspects of CRISPR/Cas for in Vivo Genome Editing
- (2019) Danny Wilbie et al. ACCOUNTS OF CHEMICAL RESEARCH
- Nose to brain delivery
- (2019) Ijeoma Uchegbu et al. JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS
- CRISPR-Cas9 system: A new-fangled dawn in gene editing
- (2019) Darshana Gupta et al. LIFE SCIENCES
- The Development of Functional Non-Viral Vectors for Gene Delivery
- (2019) Patil et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison
- (2018) Julie B Eisengart et al. GENETICS IN MEDICINE
- Physicochemical properties of cationic nanoemulsions and liposomes obtained by microfluidization complexed with a single plasmid or along with an oligonucleotide: Implications for CRISPR/Cas technology
- (2018) Roselena S. Schuh et al. JOURNAL OF COLLOID AND INTERFACE SCIENCE
- In vivo genome editing of mucopolysaccharidosis I mice using the CRISPR/Cas9 system
- (2018) Roselena Silvestri Schuh et al. JOURNAL OF CONTROLLED RELEASE
- The role of innate immunity in mucopolysaccharide diseases
- (2018) Helen Parker et al. JOURNAL OF NEUROCHEMISTRY
- Hematopoietic cell transplantation for severe MPS I in the first six months of life: The heart of the matter
- (2018) Elizabeth Braunlin et al. MOLECULAR GENETICS AND METABOLISM
- Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I
- (2017) Lalitha R. Belur et al. HUMAN GENE THERAPY
- Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9
- (2016) John G Doench et al. NATURE BIOTECHNOLOGY
- Preparation and Characterization of Chitosan Nanoparticles for Zidovudine Nasal Delivery
- (2015) Mariana da Silva Barbi et al. JOURNAL OF NANOSCIENCE AND NANOTECHNOLOGY
- Effects of Enzyme Replacement Therapy Started Late in a Murine Model of Mucopolysaccharidosis Type I
- (2015) Gabriela Pasqualim et al. PLoS One
- Enzyme replacement therapy started at birth improves outcome in difficult-to-treat organs in mucopolysaccharidosis I mice
- (2013) Guilherme Baldo et al. MOLECULAR GENETICS AND METABOLISM
- Standardization of α-L-iduronidase enzyme assay with Michaelis–Menten kinetics
- (2013) Li Ou et al. MOLECULAR GENETICS AND METABOLISM
- Neuropathology in Mouse Models of Mucopolysaccharidosis Type I, IIIA and IIIB
- (2012) Fiona L. Wilkinson et al. PLoS One
- Recombinant Encapsulated Cells Overexpressing Alpha-L-Iduronidase Correct Enzyme Deficiency in Human Mucopolysaccharidosis Type I Cells
- (2011) Guilherme Baldo et al. CELLS TISSUES ORGANS
- Real-time PCR to determine transgene copy number and to quantitate the biolocalization of adoptively transferred cells from EGFP-transgenic mice
- (2008) Molishree U. Joshi et al. BIOTECHNIQUES
- Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection
- (2008) Carmela Zincarelli et al. MOLECULAR THERAPY
- Plasmid DNA loaded chitosan nanoparticles for nasal mucosal immunization against hepatitis B
- (2007) Kapil Khatri et al. INTERNATIONAL JOURNAL OF PHARMACEUTICS
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