Integrated gene therapy successfully treated Haemophilia A in India, providing long-term relief without bleeding episodes for ...

Researchers study long-term safety of lentiviral gene therapy in primates, finding no evidence of somatic mutations or ...

The design of this lentiviral vector raises unique safety risks, because expression of the therapeutic transgene is driven by a potent murine γ-retroviral enhancer–promoter that can efficiently ...

Evercore ISI analyst Gavin Clark-Gartner maintained a Buy rating on Rocket Pharmaceuticals (RCKT – Research Report) on December 20 and set a ...

This study provides important insights into the regulation of a retained intron in the mRNA coding for OGT, a process known to be regulated by the O-GlcNAc cycling system, and highlights the ...

Hemogenyx Pharmaceuticals is a publicly traded company (LSE:HEMO) headquartered in London, with its US operating subsidiaries, Hemogenyx Pharmaceuticals LLC and Immugenyx LLC, located in New York City ...

Fintel reports that on December 18, 2024, Jefferies initiated coverage of Rocket Pharmaceuticals (NasdaqGM:RCKT) with a Buy ...

However, our understanding of how gradual changes in gene dosage affect molecular, cellular, and organismal traits is currently limited. To address this gap, we induced gradual changes in gene ...

The cull also includes Aruvant's lentiviral sickle cell disease gene therapy ARU-1801 – in trials but with questionable commercial potential given progress with rival therapies – as well as ...

在一项新的研究中，来自印度韦洛尔基督教医学院等研究机构的研究人员发现，慢病毒载体可成功用于为严重血友病A患者提供基因治疗。他们为腺相关病毒（AAV）介导的基因治疗提供了一种潜在的替代方案，解决了将事先存在抗AAV抗体的患者排除在基因治疗对象之外的问题 ...