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This invention identifies a mechanism for pausing development of pre-implantation embryos while retaining viability.

Targeted Intracellular Delivery of Nucleic Acids via Conjugation to Non-Lipid Carrier Molecules

Use of synthetic nucleic acids to manipulate gene function has become a powerful tool for both basic research and therapeutics.  Silencing disease targets by RNA interference is a promising approach to drug development, and various experimental RNA therapies are currently in clinical development by both small and large biotechnology companies.  miRNAs are also being developed for disease treatment and diagnosis.  However, lack of specifically targeted, efficient and safe vehicles for systemic delivery of small RNA payloads in vivo is a serious challenge.  Synthetic nucleic acids face a number of physiological barriers in the bloodstream, and their intracellular uptake is hampered by the fact that they are highly charged and have much larger molecular wieght than small-molecule drugs.  Current strategies to circumvent these problems includes local administration, chemical modifications of nucleic acids, viral delivery vectors, lipid-based delivery systems, polymer-based delivery systems and nanoparticle encapsulation.  These methods have serious flaws including toxicity, inummue effects, non-selectively and high cost of manufacturing.  Therefore, novel ways to deliver synthetic nucleic acids for use in humans and experimental animal models are sorely needed.

Efficient genetic system for high throughput screening of new compounds that modulate activity of potassium ion channels

Researchers at UCSF have developed a novel and efficient genetic high throughput screening system for discovery of small molecule modulators that either activate or inhibit K2P potassium channel activity. Such modulators could be used for treating diseases such as chronic pain, depression, and also to modulate responses to general anesthesia.

Carcinogenesis Model Encompassing the Range of Prostate Cancer Progression and Metastasis

Currently there is a lack of information and models for understanding human prostate cancer progression. Most models currently available only allow for comparison of tumorigenic versus non-tumorigenic states. UCSF investigators have developed a series of human prostatic epithelial cell lines that encompass the range of prostate cancer progression. These cells are derived from the parental BPH-1 non-tumorigenic immortalized human prostatic epithelial cell line (see References below) using tissue recombination methods. Upon hormonal treatment, the cells exhibited either non-tumorigenicity, tumorigenicity, epithelial to mesenchymal transition (EMT), and metastasis. Progression uniquely occurs in initiated but non-tumorgenic epithelial cells and has been characterized by histopathological criteria, tumor mass size, and associated changes in expression of gene products.

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