webinar
Power up your LNP and AAV sample prep with Unagi
12 October 2022 | By Unchained Labs
Watch this on-demand webinar to see first-hand data results demonstrating the power and flexibility of Unagi for nucleic acids, LNPs and AAVs.
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Gene Therapy
Gene Therapy is the introduction of normal genes into cells in place of missing or defective ones in order to correct genetic disorders.
news
Improved AAV could cross blood-brain barrier and deliver gene therapies to CNS
A newly developed AAV has shown promise at delivering gene therapies to the brain in mice and non-human primates.
whitepaper
Product hub: CD34+ haematopoietic stem cells: One cell type, multiple viable sources
Learn how you can mobilise CD34+ haematopoietic stem cells and how they can be isolated immediately after collection.
news
Researchers develop NPHP5 gene therapy for rare ciliopathy
The researchers found that augmenting NPHP5 gene rescues cilia defects in light-sensing cells derived from ciliopathy patients.
news
Researchers find that CRISPR gene editing can give rise to cell toxicity and genomic instability
Using computational methods, researchers analysed the most popular CRISPR library designed for human cells and found 3,300 targeted spots that show strong toxic effects.
podcasts
Episode 11 – CRISPR with Dr Jakob Haaber, SNIPR Biome & Dr Richard Fox, Infinome Biosciences
Join leading experts as they discuss the wide range of uses for CRISPR, including for therapeutics and biomanufacturing.
news
Gene therapy approach shows promise in treating ALS
In rodent models of ALS, the introduced neuroprotective protein slowed disease progression and increased life span.
article
ELRIG: New modalities in pharmacology and drug discovery
Drug Target Review’s Ria Kakkad recently visited ELRIG and the British Pharmacological Society’s conference on New Modalities in Pharmacology and Drug Discovery which took place in London, UK. This article shares some of the key takeaways from the event.
news
Concerns for future gene therapy testing in laboratory mice
Researchers have found that new sickle cell disease gene therapies depend on choosing the right laboratory mice.
news
Dysfunctional gene leads to potentially treatable deafness
Researchers have found that inherited mutations in the MINAR2 gene causes deafness. The findings suggest that deafness could be treated with genetic therapies.
news
An effective and error-free gene editor
Scientists have developed a new gene editor which uses a modified pair of molecular scissors that make nicks on opposite strands of the DNA at two different points.
news
New gene therapy improvement has massive potential
Scientists have developed a technology that can improve targeting specific organs and tissue types in gene therapy.
article
Providing new hope for rare disease using non-viral gene therapy
Viral vectors to deliver gene therapies are utilised by clinically approved therapies. However, this method is not the only option for advanced therapeutics. In this piece, Brent Warner, President, Gene Therapy at Poseida Therapeutics, discusses the balance between efficacy and safety in gene therapy, highlighting positive pre-clinical data for non-viral…
article
Challenges and opportunities: lessons learned in developing potential gene therapy cures
While the full potential of gene therapies continues to be realised, their successes in rare diseases promote them as a promising treatment modality. Dr Gaurav Shah, Chief Executive Officer of Rocket Pharmaceuticals discusses the future of gene therapies as well as the company’s progress in in vivo and ex vivo programmes.
article
Leveraging a dual technology platform to develop gene therapies for a range of rare diseases
In this article, Dr Mary Spellman, Chief Medical Officer and Senior Vice President of R&D at Castle Creek Biosciences, discusses how a versatile dual platform of ex vivo and in vivo gene therapy technologies is being utilised for the development of novel gene therapies for a broad range of rare…
