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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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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.
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Researchers use brain organoids to identify neural abnormalities and test gene therapy tools
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.
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Study shows muscular dystrophies can be repaired with CRISPR-Cas9
Researchers have introduced a CRISPR-Cas9 tool into human muscle stem cells using mRNA, thus discovering a suitable method to treat muscular dystrophies.
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New CRISPR gene-editing technology could lead to better treatments for HIV
Scientists at Northwestern Medicine have developed new techniques in human blood to pave potential paths towards a HIV cure.
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Novel genome-wide approach to engineer more effective immunotherapy
Scientists have developed a genetic screening platform to identify genes that can enhance immune cells to make them more persistent and increase their ability to eradicate tumour cells.
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Research collaboration to study ageing reversal in liver tissue established
Genflow have announced a scientific research collaboration with the University of Rochester’s Aging Research Center. Together, they will study the ageing process in liver tissue.
