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Replacing the PKP2 gene prolongs survival in ARVC patients
The new study suggests that this gene therapy method may combat ARVC in both early and more advanced stages of the condition.
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Rocket Pharmaceuticals
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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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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.