Virus-like particle mediated CRISPR/Cas9 delivery for efficient and safe genome editing
The discovery of designer nucleases has made genome editing much more efficient than
before. The designer nucleases have been widely used for mechanistic studies, animal …
before. The designer nucleases have been widely used for mechanistic studies, animal …
CRISPR-Cas9-mediated gene therapy in neurological disorders
L Guan, Y Han, C Yang, S Lu, J Du, H Li, J Lin - Molecular Neurobiology, 2022 - Springer
Neurological disorders are primarily diseases with sophisticated etiology that are always
refractory and recrudescent. The major obstruction to effective therapies for neurological …
refractory and recrudescent. The major obstruction to effective therapies for neurological …
Engineered extracellular vesicles as versatile ribonucleoprotein delivery vehicles for efficient and safe CRISPR genome editing
Transient delivery of CRISPR‐based genome editing effectors is important to reduce off‐
target effects and immune responses. Recently extracellular vesicles (EVs) have been …
target effects and immune responses. Recently extracellular vesicles (EVs) have been …
Lentiviral Capsid-Mediated Streptococcus pyogenes Cas9 Ribonucleoprotein Delivery for Efficient and Safe Multiplex Genome Editing
Transient expression of the CRISPR-Cas9 machinery is desirable to reduce the risks of off-
targets and immune responses. Electroporation of Cas9 ribonucleoproteins (RNPs) is the …
targets and immune responses. Electroporation of Cas9 ribonucleoproteins (RNPs) is the …
Targeting DNA polymerase to DNA double-strand breaks reduces DNA deletion size and increases templated insertions generated by CRISPR/Cas9
Most insertions or deletions generated by CRISPR/Cas9 (clustered regularly interspaced
short palindromic repeats/CRISPR-associated protein 9) endonucleases are short (< 25 bp) …
short palindromic repeats/CRISPR-associated protein 9) endonucleases are short (< 25 bp) …
New advances in using virus-like particles and related technologies for eukaryotic genome editing delivery
P Lyu, B Lu - International Journal of Molecular Sciences, 2022 - mdpi.com
The designer nucleases, including Zinc Finger Nuclease (ZFN), Transcription Activator-Like
Effector Nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic …
Effector Nuclease (TALEN), and Clustered Regularly Interspaced Short Palindromic …
Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy
C Sun, C Serra, BH Kalicharan, J Harding, M Rao - Cells, 2024 - mdpi.com
Cell therapies derived from induced pluripotent stem cells (iPSCs) offer a promising avenue
in the field of regenerative medicine due to iPSCs' expandability, immune compatibility, and …
in the field of regenerative medicine due to iPSCs' expandability, immune compatibility, and …
Duchenne muscular dystrophy animal models
TV Egorova, II Galkin, YV Ivanova… - … Animal Modeling in …, 2022 - books.google.com
Duchenne muscular dystrophy is a complex and severe orphan disease. It develops when
the organism lacks the expression of dystrophin-a large structural protein. Dystrophin is …
the organism lacks the expression of dystrophin-a large structural protein. Dystrophin is …
[HTML][HTML] Genetically modified animal models of hereditary diseases for testing of gene-directed therapy
AV Polikarpova, TV Egorova… - Research Results in …, 2022 - cyberleninka.ru
Disease-causing genes have been identified for many severe muscular and neurological
genetic disorders. Advances in the gene therapy field offer promising solutions for drug …
genetic disorders. Advances in the gene therapy field offer promising solutions for drug …
RESEARCH RESULTS IN PHARMACOLOGY
AV POLIKARPOVA, TV EGOROVA… - RESEARCH RESULTS …, 2022 - elibrary.ru
Disease-causing genes have been identified for many severe muscular and neurological
genetic disorders. Advances in the gene therapy field offer promising solutions for drug …
genetic disorders. Advances in the gene therapy field offer promising solutions for drug …