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Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
Cell and gene therapy is rapidly transforming the treatment of complex diseases, yet scaling production efficiently remains a challenge. Dr Tia Harmon from PHC Corporation of North America explores the innovations that are optimising cell expansion to enhance the discovery of advanced therapies.
Japanese researchers have developed a new enzyme technology that can precisely alter the levels of mutated mitochondrial DNA in patient-derived stem cells, offering a promising new approach for treating mitochondrial disorders.
Huntington's disease remains a major challenge, but allele-selective gene editing offers new hope. By targeting only the mutant gene, this approach could provide a one-time, durable treatment. Life Edit’s Dr Amy Pooler reveals how this innovative therapy could reshape HD treatment.
Outdated clinical development systems are slowing breakthroughs like gene therapies and precision medicine. This article explores how a flexible, on-demand approach can accelerate drug development and bring innovations to patients faster.
Claudia Zylberberg, Chair of Kosten Digital, reveals how AI is revolutionising cell and gene therapy, making drug discovery faster and more cost-effective.
29 April 2025 | By LevitasBio
Cell prep is a bottleneck. It’s time to rethink the workflow.
Discover how Chromatin Bioscience’s synthetic promoters are transforming gene therapy by enhancing precision, boosting safety and improving manufacturing efficiency.
Learn how to simplify the entire DNA template generation workflow, streamlining mRNA therapeutic research.
A preclinical study conducted by researchers at Weill Cornell Medicine have demonstrated a new gene-editing strategy to treat short bowel syndrome (SBS), a life-threatening condition in which patients lack a functional small intestine.
7 April 2025 | By Takara Bio USA
Use this lentiviral gene delivery workflow poster to understand how to optimize your workflow for successful viral transduction.
Gene silencing offers a promising approach to treating rare neurological diseases like H-ABC. With clinical trials on the horizon, find out how targeted therapies could bring real hope to patients and families.
21 March 2025 | By Abzena
Watch our webinar with Abzena’s experts to explore practical solutions for overcoming chemistry, manufacturing and regulatory challenges in antibody-oligonucleotide conjugate (AOC) development, enabling progress for previously untreatable diseases.
ElevateBio is teaming up with Amazon Web Services (AWS) to advance CRISPR gene editing using AI and cloud computing. This collaboration aims to accelerate drug discovery for genetic diseases, making next-generation therapies more efficient and accessible.
This Friday, February 28, marks Rare Disease Day 2025, a global initiative raising awareness for the 300 million people living with rare conditions. Dr David Reynolds, CEO of LoQus23 Therapeutics, explores how advancements in rare disease research are driving the development of new treatments.
Researchers have identified a small gene, SCN10a-short, that could enable gene therapy to treat malignant cardiac arrhythmias. This discovery offers the potential for a one-time treatment, reducing the reliance on lifelong medication and invasive procedures.
