In some aspects, the disclosure relates to compositions and methods for modulating (e.g., increasing and/or decreasing) bone mass in a subject. In some aspects, the disclosure provides isolated nucleic acids, and vectors such as rAAV vectors, configured to express transgenes that promote (e.g., increase) or inhibit (e.g., decrease) activity, differentiation, or function of certain types of bone cells, for example osteoblasts, osteoclasts, osteocytes, etc. In some embodiments, the isolated nucleic acids and vectors described by the disclosure are useful for treating disorders and conditions associated with increased bone mass (e.g., osteopetrosis) or decreased bone mass (e.g., osteoporosis).
Compositions And Methods For Transient Delivery Of Nucleases
The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AA V capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
Self-Regulating Aav Vectors For Safe Expression Of Mecp2 In Rett Syndrome
- Boston MA, US Guangping GAO - Westborough MA, US Michael R. GREEN - Boylston MA, US Dan WANG - Belchertown MA, US Tessa Mercedes SIMONE - Worcester MA, US
Assignee:
University of Massachusetts - Boston MA
International Classification:
C12N 15/86 C07K 14/47
Abstract:
In some aspects, the disclosure relates to compositions and methods of engineering a transgene. In some embodiments, the disclosure provides self-regulating recombinant nucleic acids, viral vectors and pharmaceutical compositions comprising a MeCP2 transgene. In some embodiments, compositions and methods described by the disclosure are useful for treating diseases and disorders associated with a loss of function mutation, for example Rett syndrome.
Compositions And Methods For Transient Delivery Of Nucleases
The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AAV capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
Apr 2011 to 2000 Senior Statistician & BioinformaticianBioinformatics Division
Sep 2009 to Apr 2011 AnalystBioinformatics Division Buffalo, NY Jul 2008 to Aug 2009 Graduate Research AssistantHigh School 110
Aug 2002 to Nov 2004
Education:
The State University of New York, University at Buffalo Buffalo, NY 2012 Ph.D. in BiostatisticsThe State University of New York, University at Buffalo Buffalo, NY 2007 to 2009 M.A. in BiostatisticsHuazhong Normal University Wuhan, CN 1998 to 2002 B.S. in Mathematics Education
Authors:The other authors of the paper were Shweta Gupta, Dan Wang, Francesca Marino, Arturo J. Moreno and Barbara Panning of UCSF and Yu Huang and Deepak Srivastava of UCSF and the Gladstone Institutes.
Date: Jan 22, 2025
Category: Health
Source: Google
Teardown of Huawei flagship phone finds US parts despite blacklisting
wei has shown resilience by replacing many US components over the course of a single phone-design cycle. Its continued use of Qorvo and Skyworks chips also shows that its too difficult to break dependence on US technology, said Dan Wang, technology analyst at research firm Gavekal Dragonomics.
Date: Mar 31, 2020
Category: Technology
Source: Google
China sees fall in coronavirus deaths but WHO urges caution
The employment situation is OK in the first quarter, but if the virus is not contained by end-March, then from the second quarter, well see a big round of layoffs, said Dan Wang, an analyst with the Economist Intelligence Unit (EIU). Job losses could run as high as 4.5 million, he forecast.
Date: Feb 17, 2020
Category: World
Source: Google
Doklam Standoff Between China and India Ends with Troops and Equipment Withdrawn
China analyst Dan Wang of the Economic Intelligence Unit warned CNN that the risk of both sides getting back to a military standoff is not eliminated and that some form of economic warfare might break out even if military confrontations are avoided.