Preclinical alternative drug discovery programs for monogenic rare diseases. Should small molecules or gene therapy be used? The case of hereditary spastic paraplegias

Update your browser to view ScienceDirect.

Request details:

Request ID: 8c29594b2d58270e-LAX
IP: 101.37.83.79
UTC time: 2024-09-13T16:00:12+00:00
Browser: Mozilla/5.0 (X11; U; Linux x86_64; en-US; rv:1.9.1.4) Gecko/20091030 Gentoo Firefox/3.5.4