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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.
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…
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.
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…
This podcast features an exploration of detecting host cell residual DNA in AAV vector production for gene therapy.
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.
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.
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.
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.
Researchers have found that restoring lost gene activity prevents many disease signs in an animal model of Pitt-Hopkins syndrome.
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.
Researchers have identified distinct differences among the cells comprising a tissue in the retina, findings that could help develop precise therapies for retinal diseases.
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.
Scientists have developed CRISPR with a new process that evaluates the impacts of gene edits that alter rather than deleting DNA code.
Scientists used human brain organoids to reveal how a genetic mutation associated with a profound form of autism disrupts neural development, and gene therapy tools to recover the gene’s function effectively rescued neural structure and function.
Researchers have introduced a CRISPR-Cas9 tool into human muscle stem cells using mRNA, thus discovering a suitable method to treat muscular dystrophies.
