All Gene Therapy articles – Page 3
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NewsNew enzyme tech offers hope for mitochondrial disease treatment
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.
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ArticleAllele-selective gene editing: a breakthrough in Huntington's disease treatment
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.
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ArticleBuilding the requisite clinical development infrastructure for medical innovation to thrive
Outdated clinical development systems are slowing breakthroughs like gene therapies and precision medicine.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.
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ArticleAI in drug discovery: faster, smarter, better
Claudia Zylberberg, Chair of Kosten Digital, reveals how AI is revolutionising cell and gene therapy, making drug discovery faster and more cost-effective.
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ArticleSynthetic promoters enhance gene therapy precision
Discover how Chromatin Bioscience’s synthetic promoters are transforming gene therapy by enhancing precision, boosting safety and improving manufacturing efficiency.
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WhitepaperGuide: Create DNA Template for In Vitro mRNA Synthesis
Learn how to simplify the entire DNA template generation workflow, streamlining mRNA therapeutic research.
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NewsReprogramming gut cells to treat short bowel syndrome
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.
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WhitepaperPoster: Lentiviral Gene Delivery Workflow
Use this lentiviral gene delivery workflow poster to understand how to optimize your workflow for successful viral transduction.
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ArticleGene silencing: a step forward for rare disease therapy
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.
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WebinarSolving the AOC puzzle: Strategies for chemistry, manufacturing and regulatory success
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.
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NewsAWS and ElevateBio join forces to advance gene editing
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.
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NewsRare diseases, big advances: how research is powering a new era of treatments
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.
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NewsNew gene identified for treating malignant cardiac arrhythmias
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.
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NewsDNA repair genes drive Huntington's disease, UCLA study finds
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.
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ArticleEmpowering women in STEM: championing change in genetics
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.
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ArticleExperts explore the future of iPSC-based cell therapies
Discover the transformative potential of iPSC-based therapies in regenerative medicine, alongside their challenges including scalability, safety and targeted delivery.
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NewsTLE6 protein deficiency linked to male infertility
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.
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NewsRett syndrome discovery could lead to better treatments
Researchers have identified early molecular changes in Rett syndrome that could lead to improved treatments for the condition.
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NewsStudy reveals how neurotransmitters influence brain gene expression
A new study demonstrates how neurotransmitters affect brain gene expression, offering potential therapeutic insights for circadian rhythm disorders.
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NewsNew genetic mechanism could lead to RNA therapies for mental health
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.