Home / Seminar & Event /Past Seminars / (296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
Home / Seminar & Event /Past Seminars / (296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
(296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
Seminar: (296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
Speaker: Prof. LU Boxun
Time: 2017-10-14 11:00 to 2017-10-14 12:00
Venue: Meeting room (406), Building 24
Organizer:

Health Science Platform


Abstract: Classical targeted-drug discovery is based on targeting druggable proteins, typically kinases and receptors of which the function can be agonized or antagonized. This strategy meets difficulties in cases like Huntington’s disease (HD) and similar neurodegenerative disorders, where the disease-causing proteins’ pathological function is not clear. HD is a neurodegenerative order caused by mutant HTT protein (mHTT) containing an expanded polyQ repeat stretch, but the function of mHTT and how mHTT causes HD are unknown; this prevents efforts to screen for mHTT “inhibitors”. On the other hand, HD is appealing for drug discovery because the genetics is crystal clear, as compared to other major neurodegenerative disorders, such as Alzheimer’s and Parkinson’s – where most patients present with sporadic onset. While mHTT is not directly “druggable” per se due to the unclear physiological and pathological functions, one approach that appears promising is lowering the level of the disease-causing protein, and this approach may additionally be applicable in other neurodegenerative disorders and proteinopathies linked to accumulation of aberrant proteins. Here I will discuss some of our recent target validation efforts focusing on regulation of disease protein degradation, which might provide promising revenues for drugging such diseases.


Boxun Lu_CV_title_abstract.docx


(296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
Seminar: (296) Drugging the undruggable: targeting Huntington’s Disease by modulating the disease protein levels
Speaker: Prof. LU Boxun
Time: 2017-10-14 11:00 to 2017-10-14 12:00
Venue: Meeting room (406), Building 24
Organizer:

Health Science Platform


Abstract: Classical targeted-drug discovery is based on targeting druggable proteins, typically kinases and receptors of which the function can be agonized or antagonized. This strategy meets difficulties in cases like Huntington’s disease (HD) and similar neurodegenerative disorders, where the disease-causing proteins’ pathological function is not clear. HD is a neurodegenerative order caused by mutant HTT protein (mHTT) containing an expanded polyQ repeat stretch, but the function of mHTT and how mHTT causes HD are unknown; this prevents efforts to screen for mHTT “inhibitors”. On the other hand, HD is appealing for drug discovery because the genetics is crystal clear, as compared to other major neurodegenerative disorders, such as Alzheimer’s and Parkinson’s – where most patients present with sporadic onset. While mHTT is not directly “druggable” per se due to the unclear physiological and pathological functions, one approach that appears promising is lowering the level of the disease-causing protein, and this approach may additionally be applicable in other neurodegenerative disorders and proteinopathies linked to accumulation of aberrant proteins. Here I will discuss some of our recent target validation efforts focusing on regulation of disease protein degradation, which might provide promising revenues for drugging such diseases.


Boxun Lu_CV_title_abstract.docx