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.
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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
New gene therapy improvement has massive potential
Scientists have developed a technology that can improve targeting specific organs and tissue types in gene therapy.
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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.
news
Study suggests postnatal gene therapy could treat Pitt-Hopkins syndrome
Researchers have found that restoring lost gene activity prevents many disease signs in an animal model of Pitt-Hopkins syndrome.
news
CRISPR-dCas9 gene editing reverses brain genetic reprogramming caused by adolescent binge drinking
A new study in animals has shown that gene editing may be a potential treatment for anxiety and alcohol use disorder in adults who were exposed to binge drinking in their adolescence.
news
Retinal cell map could advance precise cell and gene therapies for retinal diseases
Researchers have identified distinct differences among the cells comprising a tissue in the retina, findings that could help develop precise therapies for retinal diseases.
news
Gene therapy: A potential cure for growth-hormone resistant dwarfism
Researchers have shown that gene therapy using a single-dose injection of a virus carrying the ‘good’ gene could possibly be used to cure growth-hormone resistant dwarfism.
news
Using CRISPR to improve lung cancer treatments
Scientists have developed CRISPR with a new process that evaluates the impacts of gene edits that alter rather than deleting DNA code.