Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy
AK Dubey, E Mostafavi - Frontiers in Chemistry, 2023 - frontiersin.org
The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …
holds tremendous potential. This innovative approach combines the advantages of …
CRISPR/Cas9 delivery strategies with engineered extracellular vesicles
Therapeutic genome editing has the potential to cure diseases by directly correcting genetic
mutations in tissues and cells. Recent progress in the clustered regularly interspaced short …
mutations in tissues and cells. Recent progress in the clustered regularly interspaced short …
Current and prospective strategies for advancing the targeted delivery of CRISPR/Cas system via extracellular vesicles
X Huang, A Li, P Xu, Y Yu, S Li, L Hu… - Journal of …, 2023 - Springer
Extracellular vesicles (EVs) have emerged as a promising platform for gene delivery owing
to their natural properties and phenomenal functions, being able to circumvent the …
to their natural properties and phenomenal functions, being able to circumvent the …
CRISPR/Cas9: targeted genome editing for the treatment of hereditary hearing loss
Hereditary hearing loss (HHL) is a neurosensory disorder that affects every 1/500 newborns
worldwide and nearly 1/3 people over the age of 65. Congenital deafness is inherited as …
worldwide and nearly 1/3 people over the age of 65. Congenital deafness is inherited as …
Functionalized extracellular nanovesicles as advanced CRISPR delivery systems
The clustered regularly interspaced short palindromic repeat (CRISPR) system, an emerging
tool for genome editing, has garnered significant public interest for its potential in treating …
tool for genome editing, has garnered significant public interest for its potential in treating …
Characterization of promoters for adeno-associated virus mediated efficient Cas9 activation in adult Cas9 knock-in murine cochleae
X Zhao, C Jin, T Dong, Z Sun, X Zheng, B Feng… - Hearing Research, 2020 - Elsevier
CRISPR/Cas9 gene editing enables the treatment of hearing loss in congenitally deaf
neonatal mice via both viral and non-viral delivery. While adeno-associated virus (AAV) …
neonatal mice via both viral and non-viral delivery. While adeno-associated virus (AAV) …
Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping
P Gee, MSY Lung, Y Okuzaki, N Sasakawa… - Nature …, 2020 - nature.com
Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may
cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is …
cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is …
Simple in Vivo Gene Editing via Direct Self-Assembly of Cas9 Ribonucleoprotein Complexes for Cancer Treatment
SM Kim, SC Shin, EEK Kim, SH Kim, K Park, SJ Oh… - ACS …, 2018 - ACS Publications
Cas9 ribonucleoprotein (RNP)-mediated delivery has emerged as an ideal approach for in
vivo applications. However, the delivery of Cas9 RNPs requires electroporation or lipid-or …
vivo applications. However, the delivery of Cas9 RNPs requires electroporation or lipid-or …
Delivery of CRISPR/Cas9 using AAV-PHP. B in the inner ear leads to allele-specific inactivation of the mutated Tmc1 allele and protects auditory function in Beethoven …
P Solanes, S Spataro, Y Asai, B Pan… - Human Gene …, 2019 - infoscience.epfl.ch
Hearing Loss (HL) is the most prevalent sensorineural disorder, affecting 360 million people
worldwide. As genetic causes lead to 50% of pre-lingual deafness, gene therapy is …
worldwide. As genetic causes lead to 50% of pre-lingual deafness, gene therapy is …
[HTML][HTML] Small extracellular vesicles (sEVs)-based gene delivery platform for cell-specific CRISPR/Cas9 genome editing
S Dubey, Z Chen, YJ Jiang, A Talis, A Molotkov… - Theranostics, 2024 - ncbi.nlm.nih.gov
Small extracellular vesicles (sEVs) are naturally occurring vesicles that have the potential to
be manipulated to become promising drug delivery vehicles for on-demand in vitro and in …
be manipulated to become promising drug delivery vehicles for on-demand in vitro and in …