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.
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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
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…
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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.
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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…
podcasts
Episode 9 – rDNA detection with Dr Jorge Haller, Prevail Therapeutics & Dr Mark White, Bio-Rad
This podcast features an exploration of detecting host cell residual DNA in AAV vector production for gene therapy.
whitepaper
Application note: Advancing CAR T-cell therapy with CTS TrueCut Cas9 Protein
Read this free application note where we look at the CTS TrueCut Cas9 Protein, from its detailed quality specifications to its performance in primary T cells.
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Drug Target Review Cell & Gene Therapy ebook
This ebook features articles from gene therapy specialists that look at current platforms as well as the future of promising modalities for gene therapies. It also tackles novel stem cell technology considerations and gives insight into the careful balancing act between efficacy and safety in gene therapy.
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Drug Target Review – Issue 2 2022
In this issue are articles focusing on a novel stem cell therapy for hearing loss, how a non-linear chiroptical effect could aid drug discovery and the advantages of cryo-electron tomography for imaging research. Also included are pieces on gene therapy, spheroids and genomics.
news
Mouse study shows gene therapy could fight against rare disease
A new study shows that gene therapy could correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements, and recurrent seizures.