Results 111 to 120 of about 176,088 (291)

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, EarlyView.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Pyramiding Mn-superoxide dismutase transgenes to improve persistence and biomass production in alfalfa [PDF]

, 2002
Karen E. Samis
openalex   +1 more source

Adaptive Machine Learning Framework for Optimizing the Affinity Purification of Adeno‐Associated Viral Vectors

Biotechnology and Bioengineering, EarlyView.
ABSTRACT Adeno‐associated viral (AAV) vectors for gene therapy are becoming integral to modern medicine, providing therapeutic options for diseases once deemed incurable. Currently, viral vector purification is a critical bottleneck in the gene therapy industry, impacting product efficacy and safety as well as accessibility and cost to patients ...
Kelvin P. Idanwekhai, Shriarjun Shastry, Morgan R. Hurst, Arianna Minzoni, Eduardo Barbieri, Luke Remmler, Eugene N. Muratov, Michael A. Daniele, Stefano Menegatti, Alexander Tropsha   +9 more
wiley   +1 more source

319. Improvement of Transgene Expression through the Direct Intrahepatic Injection of Adenoviral Vectors [PDF]

, 2004

openalex   +1 more source

Enhancing CHO cell recombinant protein production using a perfusion‐directed host evolution approach

Biotechnology Progress, EarlyView.
Abstract Clonally derived cell lines generated from Chinese hamster ovary (CHO) cells encounter numerous stressors when cultured in high‐intensity perfusion bioreactors leading to poor process performance. To circumvent this, the ability of CHO cells to adapt to different culture environments was exploited. Here host cells were selected in the presence
Peter Amaya, Rajesh K. Mistry, Susie Sou, Sonja Hess, Bijay Khanal, Zayla Schaeffer, Jie Zhu, Lina Chakrabarti   +7 more
wiley   +1 more source

475. Functional Redundancy of Immunomodulatory Genes: Potent Protective Immunity Is Induced Against Pseudomonas Aeruginosa through Immunization with Antigen-Pulsed Dendritic Cells Genetically Modified with Different Immunomodulatory Transgenes [PDF]

, 2002

openalex   +1 more source

Innovative methodology for flexible monitoring of various bioprocesses by using pre‐existing Raman data coupled with automated transfer learning technique

Biotechnology Progress, EarlyView.
The application of the transfer learning algorithm successfully allowed the use of a model built on process A for real‐time monitoring of process B. This study shows the improvement obtained with this methodology compared to the direct application of the model.
Adèle Schini, Hadi El Radi, Johan Cailletaud, Célia Sanchez, Nathan Gay, Bruno Ebel, Emmanuel Guedon, Laurent Jourdainne   +7 more
wiley   +1 more source

The response of microbiome assembly within different niches across four stages to the cultivation of glyphosate-tolerant and conventional soybean varieties

Frontiers in Microbiology
IntroductionPlants are inherently connected with the microbiome, which plays a crucial role in regulating various host plant biological processes, including immunity, nutrient acquisition, and resistance against abiotic and biotic stresses.
Shengqian Chao, Shengqian Chao, Shengqian Chao, Yu Sun, Yu Sun, Yu Sun, Yin Zhang, Yin Zhang, Yin Zhang, Yifan Chen, Yifan Chen, Yifan Chen, Lili Song, Lili Song, Lili Song, Peng Li, Peng Li, Peng Li, Xueming Tang, Jingang Liang, Beibei Lv, Beibei Lv, Beibei Lv, Beibei Lv   +23 more
doaj   +1 more source

The Presence of a Chromatin Boundary Appears to Shield a Transgene in Tobacco from RNA Silencing [PDF]

, 2003
Ľudmila Mlynárová, Andrea Hricová, Annelies E. H. M. Loonen, Jan‐Peter Nap   +3 more
openalex   +1 more source

Sequential, chromosome‐specific glutamine synthetase double knockout with Cas‐CLOVER establishes enhanced CHO platforms for cell line development

Biotechnology Progress, EarlyView.
This study established the first creation of a commercially available double GS knockout (DKO) from a single KO in CHO‐K1 cells, called CleanCut GS CHO using Cas‐CLOVER. When combined with the Harbor‐IN transposase system, CleanCut GS CHO cells demonstrated a highly antibody productivity.
Cintia Gomez Limia, Valeriya Steffey, Hsueh Che Cheng, Daniel Machado, Thomas Hart, Mark Cameron McHargue, Corey Brizzee, Jack Crawford   +7 more
wiley   +1 more source