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 …

CRISPR/Cas9 delivery strategies with engineered extracellular vesicles

Y Lu, K Godbout, G Lamothe, JP Tremblay - Molecular Therapy-Nucleic …, 2023 - cell.com
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 …

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 …

CRISPR/Cas9: targeted genome editing for the treatment of hereditary hearing loss

R Farooq, K Hussain, M Tariq, A Farooq… - Journal of Applied …, 2020 - Springer
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 …

Functionalized extracellular nanovesicles as advanced CRISPR delivery systems

S Wang, H Kong, C Zhuo, L Liu, S Lv, D Cheng… - Biomaterials …, 2024 - pubs.rsc.org
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 …

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) …

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 …

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 …

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 …

[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 …