Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system
Published 2022 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Brain and visceral gene editing of mucopolysaccharidosis I mice by nasal delivery of the CRISPR/Cas9 system
Authors
Keywords
-
Journal
JOURNAL OF GENE MEDICINE
Volume -, Issue -, Pages -
Publisher
Wiley
Online
2022-01-15
DOI
10.1002/jgm.3410
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- 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
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 MoreAdd your recorded webinar
Do you already have a recorded webinar? Grow your audience and get more views by easily listing your recording on Peeref.
Upload Now