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Assembly of CRISPR ribonucleoproteins with biotinylated oligonucleotides via an RNA aptamer for precise gene editing
- Jared Carlson-Stevermer, Amr A Abdeen, +4 authors K. Saha
- Biology, Medicine
- Nature Communications
- 23 November 2017
Writing specific DNA sequences into the human genome is challenging with non-viral gene-editing reagents, since most of the edited sequences contain various imprecise insertions or deletions. We… Expand
Human iPSC modeling reveals mutation-specific responses to gene therapy in Best disease
Dominantly inherited disorders are not typically considered therapeutic candidates for gene augmentation. Here, we utilized patient-specific induced pluripotent stem cell-derived retinal pigment… Expand
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing
Delivery technologies for the CRISPR-Cas9 (CRISPR, clustered regularly interspaced short palindromic repeats) gene editing system often require viral vectors, which pose safety concerns for… Expand
Manufacturing Cell Therapies Using Engineered Biomaterials.
Emerging manufacturing processes to generate regenerative advanced therapies can involve extensive genomic and/or epigenomic manipulation of autologous or allogeneic cells. These cell engineering… Expand
Versatile Redox-Responsive Polyplexes for the Delivery of Plasmid DNA, Messenger RNA, and CRISPR-Cas9 Genome-Editing Machinery.
- Y. Wang, B. Ma, +4 authors S. Gong
- Materials Science, Medicine
- ACS applied materials & interfaces
- 17 September 2018
Gene therapy holds great promise for the treatment of many diseases, but clinical translation of gene therapies has been slowed down by the lack of safe and efficient gene delivery systems. Here, we… Expand
A pH-responsive silica-metal-organic framework hybrid nanoparticle for the delivery of hydrophilic drugs, nucleic acids, and CRISPR-Cas9 genome-editing machineries.
- Yuyuan Wang, Pawan K. Shahi, +7 authors S. Gong
- Chemistry, Medicine
- Journal of controlled release : official journal…
- 4 May 2020
Efficient delivery of hydrophilic drugs, nucleic acids, proteins, and any combination thereof is essential for various biomedical applications. Herein, we report a straightforward, yet versatile… Expand
Human iPSC modeling elucidates mutation-specific responses to gene therapy in a genotypically diverse dominant maculopathy
Dominantly inherited disorders are not typically considered therapeutic candidates for gene augmentation (GA). We tested whether GA or genome editing (GE) could serve as a solo therapy for autosomal… Expand
Combinatorial screening of biochemical and physical signals for phenotypic regulation of stem cell–based cartilage tissue engineering
Multicomponent biomaterials using a high-throughput system regulate hyaline or hypertrophic chondrogenesis of hMSCs. Despite great progress in biomaterial design strategies for replacing damaged… Expand
Human iPSC Modeling Reveals Mutation-Specific Responses to Gene Therapy in a Genotypically Diverse Dominant Maculopathy.
- Divya Sinha, B. Steyer, +16 authors D. Gamm
- Biology, Medicine
- American journal of human genetics
- 20 July 2020
Dominantly inherited disorders are not typically considered to be therapeutic candidates for gene augmentation. Here, we utilized induced pluripotent stem cell-derived retinal pigment epithelium… Expand
Combinatorial screening of biochemical and physical signals for phenotypic regulation of stem cell-based cartilage tissue engineering.
Despite great progress in biomaterial design strategies for replacing damaged articular cartilage, prevention of stem cell-derived chondrocyte hypertrophy and resulting inferior tissue formation is… Expand