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Gene Replacement Therapy Articles & Analysis: This-Year
12 articles found
Fluoropolymers: A Critical Enabler in Cell and Gene Therapies Cell and gene therapies are revolutionizing medicine, offering cures for rare diseases and innovative treatments for cancer. Behind every successful therapy is a precise and sterile manufacturing process—where the choice of materials can make the difference between success and ...
Neurotrophic factors are a group of proteins that play a crucial role in the growth, survival, and differentiation of neurons in the nervous system. They serve as essential signaling molecules that not only support the development of the nervous system during embryonic stages but also maintain neuronal health throughout an individual’s life. These factors are vital for neuroplasticity, ...
In modern biopharmaceutical manufacturing, downstream processing development has become one of the most critical and resource-intensive stages. While upstream cell culture or fermentation focuses on producing the target biomolecule, downstream steps transform crude harvests into highly purified and stable therapeutic products. For biologics such as monoclonal antibodies, recombinant proteins, and ...
Virus-like particles (VLPs) have garnered significant attention in recent years for their potential applications in vaccine development, drug delivery, and as tools in various biotechnological fields. These nanoparticles mimic the structure of viruses but lack the viral genetic material that makes viruses infectious. This unique characteristic offers a versatile platform for various innovative ...
In recent years, the field of gene editing has experienced groundbreaking advancements, with CRISPR/Cas9 emerging as a powerful technology for precise genetic manipulation. Among various delivery systems, lentiviruses have garnered significant attention due to their ability to introduce genetic material into both dividing and non-dividing cells, making them an ideal vehicle for gene therapy ...
Adenoviruses are a group of viruses that have gained significant attention in recent years, particularly in the fields of gene therapy, vaccine development, and cancer treatment. With their ability to deliver genetic material into cells effectively, adenoviruses serve as valuable vectors in various biomedical applications. To harness their potential, specialized adenovirus production services ...
The BCL2 gene, short for B-cell lymphoma 2, is a critical regulator of apoptosis, or programmed cell death. It belongs to a larger BCL2 gene family, which includes both pro-apoptotic and anti-apoptotic proteins that maintain a delicate balance between cell survival and cell death. Dysregulation of this gene family is implicated in a variety of diseases, most notably cancer. What is BCL2? The ...
Podocytes are specialized epithelial cells located in the kidneys, playing a crucial role in the filtration barrier of the glomerulus. These unique cells have garnered significant attention in scientific research due to their pivotal function in maintaining kidney health and their involvement in various kidney diseases. The Structure and Function of Podocytes Podocytes possess a distinct ...
Introduction Custom knockout cell lines represent a groundbreaking advancement in molecular biology and genetics, allowing researchers to manipulate genes with precision and study their functions in a controlled environment. By creating cell lines with specific genes “knocked out,” scientists can investigate disease mechanisms, screen drug candidates, and develop gene therapies more ...
The study of cardiovascular biology is essential for understanding the mechanisms that regulate heart function and vascular health. As cardiovascular diseases remain the leading cause of death worldwide, advancements in this field offer crucial insights for developing new treatments and preventive strategies. Understanding the Cardiovascular System The cardiovascular system comprises the heart, ...
Double-stranded RNA (dsRNA) has gained significant attention in the fields of molecular biology, genetics, and therapeutics due to its pivotal role in gene silencing and the modulation of cellular processes. The production of dsRNA can be achieved through various methods, each offering unique advantages depending on the desired application. This article delves into the primary techniques for ...
Human mesenchymal stem cells (hMSCs) are a remarkable type of stem cell found in various tissues, such as bone marrow, adipose tissue, and umbilical cord. These cells have garnered significant attention in the field of regenerative medicine and therapeutic research due to their unique properties and potential applications. Characteristics of hMSCs hMSCs possess the ability to differentiate into ...