[HTML][HTML] CRISPR-Cas9: a preclinical and clinical perspective for the treatment of human diseases
G Sharma, AR Sharma, M Bhattacharya, SS Lee… - Molecular Therapy, 2021 - cell.com
At present, the idea of genome modification has revolutionized the modern therapeutic
research era. Genome modification studies have traveled a long way from gene …
research era. Genome modification studies have traveled a long way from gene …
Advances and challenges for hemophilia gene therapy
P Batty, D Lillicrap - Human molecular genetics, 2019 - academic.oup.com
Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8
(hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding …
(hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding …
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for
therapeutic genome editing. There have been several recent reports of genome editing in …
therapeutic genome editing. There have been several recent reports of genome editing in …
Cell-specific CRISPR–Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins
MD Hoffmann, S Aschenbrenner, S Grosse… - Nucleic acids …, 2019 - academic.oup.com
The rapid development of CRISPR–Cas technologies brought a personalized and targeted
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …
treatment of genetic disorders into closer reach. To render CRISPR-based therapies precise …
CRISPR technologies for stem cell engineering and regenerative medicine
CRISPR/Cas9 system exploits the concerted action of Cas9 nuclease and programmable
single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …
single guide RNA (sgRNA), and has been widely used for genome editing. The Cas9 …
CRISPR/Cas9: A powerful genome editing technique for the treatment of cancer cells with present challenges and future directions
Cancer is one of the most leading causes of death and a major public health problem,
universally. According to accumulated data, annually, approximately 8.5 million people died …
universally. According to accumulated data, annually, approximately 8.5 million people died …
Current updates of CRISPR/Cas9‐mediated genome editing and targeting within tumor cells: an innovative strategy of cancer management
Clustered regularly interspaced short palindromic repeats‐associated protein
(CRISPR/Cas9), an adaptive microbial immune system, has been exploited as a robust …
(CRISPR/Cas9), an adaptive microbial immune system, has been exploited as a robust …
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
S Dastidar, S Ardui, K Singh, D Majumdar… - Nucleic acids …, 2018 - academic.oup.com
CRISPR/Cas9 is an attractive platform to potentially correct dominant genetic diseases by
gene editing with unprecedented precision. In the current proof-of-principle study, we …
gene editing with unprecedented precision. In the current proof-of-principle study, we …
[HTML][HTML] Long-term correction of hemophilia B using adenoviral delivery of CRISPR/Cas9
CJ Stephens, EJ Lauron, E Kashentseva, ZH Lu… - Journal of controlled …, 2019 - Elsevier
Hemophilia B (HB) is a life-threatening inherited disease caused by mutations in the FIX
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …
gene, leading to reduced protein function and abnormal blood clotting. Due to its monogenic …
Approach for in vivo delivery of CRISPR/Cas system: a recent update and future prospect
YF Chuang, AJ Phipps, FL Lin, V Hecht… - Cellular and Molecular …, 2021 - Springer
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-
associated (Cas) system provides a groundbreaking genetic technology that allows …
associated (Cas) system provides a groundbreaking genetic technology that allows …