Results 121 to 130 of about 605,611 (288)

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

Monitoring pharmacodynamic and molecular drug targets in liquid biopsy: Exploratory study in liver cancer with modelling of EGFR Receptor engagement

British Journal of Clinical Pharmacology, EarlyView.
Abstract Aim Liquid biopsy is minimally invasive (compared with tissue biopsy) and has previously been used to generate systems data regarding drug elimination via hepatic enzymes and transporters. This study extends quantitative assessment of systems parameters in liquid biopsy to pharmacodynamic (PD) and disease markers relevant to cancer development
Zubida M. Al‐Majdoub, Armin Sepp, Amin Rostami‐Hodjegan, Brahim Achour   +3 more
wiley   +1 more source

Cell and Gene Therapies Manufacturing Challenges and Integrated Good Manufacturing Practices Solutions: A Lifecycle Perspective

Biotechnology and Bioengineering, EarlyView.
Lifecycle perspective on cell and gene therapy manufacturing challenges and enabling GMP solutions. ABSTRACT Cell and gene therapies (CGTs) are revolutionizing modern medicine; however, making these advanced medicines scalable and readily available to commercial manufacturers worldwide is a major challenge. The number of approved CGT products continues
Rajath Samaga, Udisha Singh, Sachin Dattram Pawar   +2 more
wiley   +1 more source

Targeting neutrophil extracellular traps in metabolic and immune niche: Nanomaterials for diabetes tissue regeneration

BMEMat, EarlyView.
The effects of NETs on regeneration of various diabetic tissues, and strategies targeting NETs for diabetes tissue regeneration. In the diabetic environment, NETs undergo complex metabolic and immune reprogramming, leading to dynamic changes in antibacterial and proinflammatory functions, and affecting regeneration of multiple systemic tissues.
Xinyi Jiang, Muxin Yue, Kai Mao, Boon Chin Heng, Noor Azlin Yahya, Yunsong Liu, Zheng Li   +6 more
wiley   +1 more source

Neurovascular coupling in bone regeneration: Mechanisms, advanced biomaterials and challenges

BMEMat, EarlyView.
This figure illustrates various material strategies for neurovascularized bone regeneration, including electroactive scaffolds, ion‐loaded materials, drug delivery systems, surface modifications, cells/cell products, growth factors, and peptides. These approaches aim to synergistically promote the regeneration of neural, vascular, and bone tissues ...
Yixin Ma, Nan Jiang, Liyuan Chen, He Zhang, Xinjia Cai, Karan Gulati, Danqing He, Zhigang Cai, Yan Liu   +8 more
wiley   +1 more source

Metal nanostructure‐coupled label‐free localized surface plasmon resonance biosensors for rapid and sensitive disease diagnosis

BMEMat, EarlyView.
LSPR biosensing enables rapid, sensitive, and low‐sample‐volume detection of biomarkers, with broad applications in diagnosing cancer, infectious, and chronic diseases. Advances in nanostructure design, micro/nanofabrication, and microfluidic integration have expanded the potential of LSPR sensors in biomedical diagnostics.
Zhaoxin Geng, Yunfei Sha, Jiongyan Yang, Hongru Yang, Xiao Liang   +4 more
wiley   +1 more source

Feasibility of combined treatment for type III spinal muscular atrophy: a pilot study

, 2017
Nataliia Sych, 1 Mariya Klunnyk,1 Iryna Matiyashchuk,1 Mariya Demchuk,1 Olena Ivankova,1 Andriy Sinelnyk,1 Khrystyna Sorochynska,2 Marina Skalozub3 1Clinical Department, 2Stem Cells Bank, 3Laboratory and Biotechnology Department, Cell Therapy Center ...
Sych N, Matiyashchuk I, Sorochynska K, Klunnyk M, Sinelnyk A, Skalozub M, Ivankova O, Demchuk M   +7 more
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Status and future of recombinant adeno‐associated virus vector manufacturing

Biotechnology Progress, EarlyView.
Abstract Sixty years of adeno‐associated virus (AAV) research illustrates a trajectory marked by basic science exploration, iterative innovation, persistent challenges, a number of clinical setbacks, as well as commercial therapeutic triumphs. This continual evolution has led to recombinant AAV (rAAV) becoming a cornerstone of modern gene therapy ...
Frank Agbogbo, David Dismuke
wiley   +1 more source

A novel case report of spinal muscular atrophy with progressive myoclonic epilepsy from Iran

, 2019
Reza Shervin Badv,1 Yalda Nilipour,2 Shahram Rahimi-Dehgolan,3 Ali Rashidi-Nezhad,4 Masood Ghahvechi Akbari51Children’s Medical Center, Pediatrics Center of Excellence, Tehran University of Medical Sciences (TUMS), Tehran, Iran; 2Pediatric ...
Shervin Badv R, Ghahvechi Akbari M, Rahimi-Dehgolan S, Rashidi-Nezhad A, Nilipour Y   +4 more
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Novel capillary defects in spinal muscular atrophy [PDF]

, 2015
Spinal Muscular Atrophy (SMA) is an autosomal, recessive form of childhood motor neuron disease and the most common genetic cause of infant mortality in the western world.
Somers, Eilidh
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