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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.
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
Join 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.
A new UCLA study reveals that DNA mismatch repair genes play a crucial role in Huntington’s disease by driving neuronal damage and motor impairments. Targeting these genes, especially Msh3 and Pms1, could offer promising therapeutic avenues for the disease.
An advocate for genetic medicines and the enormous potential they hold, Leah Sabin at Regeneron Genetic Medicines reveals how passion and curiosity can forge the path to a rewarding career in STEM.
19 February 2025 | By
Discover the transformative potential of iPSC-based therapies in regenerative medicine, alongside their challenges including scalability, safety and targeted delivery.
New study reveals that TLE6 protein deficiency causes male infertility in mice. These findings suggest potential genetic causes and future treatment avenues for male infertility.
Researchers have identified early molecular changes in Rett syndrome that could lead to improved treatments for the condition.
A new study demonstrates how neurotransmitters affect brain gene expression, offering potential therapeutic insights for circadian rhythm disorders.
Researchers at the Medical University of South Carolina have discovered a key genetic mechanism that could lead to RNA-based therapies for psychiatric disorders triggered by emotional experiences.
The LiCellGrow cell expansion system enables real-time metabolic monitoring and automated culture optimisation to accelerate cell and gene therapy manufacturing.
Find out how Tessera's latest advancements in gene editing bring promising solutions to treat sickle cell disease.
Find out how in vivo cell therapies could transform cancer treatment by providing faster, more affordable, and accessible options for more patients.
