25 papers · ranked by Valyu relevance
Han Zhang, Nathan Bamidele, Pengpeng Liu, Ogooluwa Ojelabi + 8 more
Base editors (BEs) have opened new avenues for the treatment of genetic diseases. However, advances in delivery approaches are needed to enable disease targeting of a broad range of tissues and cell types. Adeno-associated virus (AAV) vectors remain one of the most promising delivery vehicles for gene therapies.…
Suhani Vora, Jenny Cheng, Ru Xiao, Nathan J. VanDusen + 5 more
Akin to Zinc Finger and Transcription Activator Like Effector based transcriptional modulators, nuclease-null CRISPR-Cas9 provides a groundbreaking programmable DNA binding platform, begetting an arsenal of targetable regulators for transcriptional and epigenetic perturbation, by either directly tethering, or…
Tyler Kozisek, Luke Samuelson, Andrew Hamann, Angela K. Pannier
Background Human mesenchymal stem cells (hMSCs) are being researched for cell-based therapies due to a host of unique properties, however, genetic modification of hMSCs, accomplished through nonviral gene delivery, could greatly advance their therapeutic potential. Furthermore, expression of multiple transgenes in…
Kiran Kaladharan, Ashish Kumar, Pallavi Gupta, Kavitha Illath + 3 more
'Tuhin Subhra Santra' 'Fan-Gang Tseng' 'Carlos Escobedo'] The ability to deliver foreign molecules into a single living cell with high transfection efficiency and high cell viability is of great interest in cell biology for applications in therapeutic development, diagnostics, and drug delivery towards personalized…
Sylwia Bobis-Wozowicz, Karolina Kania, Kinga Nit, Natalia Blazowska + 13 more
Precise genome editing using designer nucleases (DNs), such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeat/Cas9 (CRISPR/Cas9) system, has become a method of choice in a variety of biological and biomedical…
Soultana Konstantinidou, Agnieszka Lindstaedt, Tiziana Julia Nadjeschda Schmidt, Francesco Nocilla + 12 more
In recent years, the CRISPR/Cas9 technology has emerged as a highly efficient tool for cell gene editing. However, the delivery of the CRISPR/Cas9 system into cells remains a significant challenge, drastically limiting in vivo gene therapy applications. In this study, we present a transfection/transduction-free tool…
Philippe E. Mangeot, Valérie Risson, Floriane Fusil, Aline Marnef + 14 more
Programmable nucleases have enabled rapid and accessible genome engineering in eukaryotic cells and living organisms. However, their delivery into target cells can be technically challenging when working with primary cells or in vivo. Using engineered murine leukemia virus-like particles loaded with Cas9/sgRNA…
Nina Bono, Federica Ponti, Diego Mantovani, Gabriele Candiani
Transfection by means of non-viral gene delivery vectors is the cornerstone of modern gene delivery. Despite the resources poured into the development of ever more effective transfectants, improvement is still slow and limited. Of note, the performance of any gene delivery vector in vitro is strictly dependent on…
Sofía Mirón-Barroso, Elena B. Domènech, Sonia Trigueros, Thierry Bordet
'Thierry Bordet'] Nanomaterials are currently being developed for the specific cell/tissue/organ delivery of genetic material. Nanomaterials are considered as non-viral vectors for gene therapy use. However, there are several requirements for developing a device small enough to become an efficient gene-delivery tool.…
Akh, Leyla, Fajrial, Apresio K. + 12 more
Genetic engineering of cells has a range of applications in treating incurable diseases. Plasmid DNA is a popular choice of nucleic acid for cell engineering due to its low cost and stability. However, plasmid DNA must survive the protective mechanisms present in the cell's cytoplasm to enter the nucleus for…
Boris Mitavskiy, Jun He
In the current work we introduce a novel estimation of distribution algorithm to tackle a hard combinatorial optimization problem, namely the single-machine scheduling problem, with uncertain delivery times. The majority of the existing research coping with optimization problems in uncertain environment aims at finding…
Authors not listed
In this report, we examine the extensive research landscape of CRISPR with an emphasis on CRISPR therapeutics and showcase our results from an in-depth analysis of the most up-to-date scientific information consisting of more than 53,000 publications encompassing academic journal articles and patents, spanning nearly…
Giulia Bruno
| | Contents 3 | | --- | --- | | Summary | 5 | | 1 | Cell signalling and electrophysiology development 8 | | 1.1 | Basics of cell communication and its main components 8 | | 1.1.1 | Action potential 8 | | 1.1.2 | Neuron 9 | | 1.1.3 | Cardiomyocyte 11 | | 1.2 | History of electrophysiology and MEA development 11 | | 2 |…
Svitlana Gorobets, Oksana Gorobets, I. V. Sharau, Yu. V. Milenko
For decades gene or protein replacement therapy has been proposed as means of preventing and treating various human diseases, in particular cancer [1] and monogenic diseases such as hemophilia and cystic disease, fibrosis [2–5]. The delivery of proteins or nucleic acids to the target cells is carried out through…
Marina Mariconti, Virginie Escriou, Mathieu Morel, Damien Baigl + 2 more
Using functional proteins for therapeutic purposes due to their high selectivity and/or catalytic properties can enable the control of various cellular processes, however, the transport of active proteins inside living cells remains a major challenge. In contrast, intracellular delivery of nucleic acids has become a…
Zahir Ali, Maged Serag, Gozde Demirer, Bruno Torre + 4 more
Efficient delivery of DNA, RNA, and genome engineering machinery to plant cells will enable efforts to genetically modify plants for global food security, sustainable energy production, synthetic biology applications, and climate change resilience. For the delivery of functional genetic units into plant cells, charged…
Harrison Lawson, Huy Nguyen, Ayesha Tupe, Mariah Arral + 4 more
Over the last two decades, the utility of metal-organic frameworks (MOFs) has expanded from catalysis and gas storage to biomedical applications such as drug delivery. At first, MOF-based delivery platforms focused on delivering small molecules, with current work focusing on nucleic acids, such as DNA, short guide RNA…
Agnieszka Fus-Kujawa, Pawel Prus, Karolina Bajdak-Rusinek, Paulina Teper + 3 more
Transfection is a powerful analytical tool enabling studies of gene products and functions in eukaryotic cells. Successful delivery of genetic material into cells depends on DNA quantity and quality, incubation time and ratio of transfection reagent to DNA, the origin, type and the passage of transfected cells, and the…
Jonathan D. Boucher, Devin M. Stranford, Hailey I. Edelstein, Danielle Tullman-Ercek + 2 more
Adeno-associated virus (AAV) gene therapies have achieved clinical success, with multiple products reaching regulatory approval. Encapsulation of AAV vectors within engineered extracellular vesicles (EVs) is an emerging strategy which could help overcome challenges including pre-existing anti- capsid immunity and the…
Sébastien Benizri, Arnaud Gissot, Andrew Martin, Brune Vialet + 2 more
'Mark W. Grinstaff' 'Philippe Barthélémy'] ABSTRACT: Oligonucleotide-based agents have the potential to treat or cure almost any disease, and are one of the key therapeutic drug classes of the future. Bioconjugated oligonucleotides, a subset of this class, are emerging from basic research and being successfully…
Josh Smith, Sara Carillo, Aditya Kulkarni, Erin Redman + 2 more
Adeno-associated viruses (AAVs) are viral vectors used as delivery systems for gene therapies. Intact protein characterization of AAV viral capsid proteins (VPs) and their post-translational modifications is critical to ensuring product quality. In this study, microchip based ZipChip capillary electrophoresis-mass…
Takeshi Suda, Takeshi Yokoo, Tsutomu Kanefuji, Kenya Kamimura + 3 more
'Guisheng Zhang' 'Dexi Liu' 'Dimitrios A. Lamprou'] The principle of hydrodynamic delivery was initially used to develop a method for the delivery of plasmids into mouse hepatocytes through tail vein injection and has been expanded for use in the delivery of various biologically active materials to cells in various…
Janet Sasso, Barbara Ambrose, Rumiana Tenchov, Ruchira Datta + 3 more
In the last decade, there has been a shift in research, clinical development, and commercial activity to exploit the many roles of RNA in physiology for use in medicine. With the rapid success in the development of lipid-RNA nanoparticles for mRNA vaccines against COVID-19 and with several approved RNA-based drugs, RNA…
Anna G Manjón, Hans Teunissen, Elzo de Wit, René H Medema
The discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR) and its development as a genome editing tool has revolutionized the field of molecular biology. In the DNA damage field, CRISPR has brought an alternative to induce endogenous double-strand breaks (DSB) at desired genomic locations…
Weilu Shen, Anqi Chen, Gurminder K. Paink, Nicole Black + 2 more
'David A. Weitz' 'Eric Mazur'] Abstract: Effective intracellular delivery is essential for successful gene editing of cells. Spatially selective delivery to cells that is simultaneously precise, consistent, and nondestructive remains challenging using conventional state-of-the-art techniques. Here, we introduce a…