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Showing 5 matches for the following: Inventor: "Cullen, Bryan" ×
Gene therapy for long-term treatment of GSDIa
Unmet Need Glycogen storage disease type Ia (GSDIa), or von Gierke disease, is a rare heritable metabolic disorder that impacts the liver and kidneys due to an inability to break down glycogen. An estimated 1…
Method for increasing gene expression in mammalian cells from transfected plasmids
Unmet Need Mammalian cell-based protein expression systems enable the production of mammalian proteins in the most native structure and activity. Thus, they have been commonly used for producing antibodies and therapeutic proteins, and more recently,…
Inactivating viral replication with CRISPR/Cas systems
Value Proposition Current gene therapy approaches based upon targeted DNA endoculeases, such as zinc finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), are based upon custom built DNA binding domains. Although gene therapy with…
Viral microRNAs expressed in cells latently infected with Herpes Simplex Virus Type 1
The present invention relates, in general, to micro RN As and, in particular, to viral microRNAs expressed by Herpes Simplex Vims 1 (HSV-1) or Herpes Simplex Virus 2 (HSV-2), to agents that inhibit such microRNAs…
Selective inhibition of mammalian gene expression by endogenous production of designed micro RNAs (miRNAs)
This patent pending technology uses artificial miRNAs, transcribed from a plasmid or viral vector, to specifically and stably eliminate protein expression for individual gene targets both in vitro and in vivo. The unique temporal and/or…