Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387296965> ?p ?o ?g. }
- W4387296965 abstract "Spinal muscular atrophy (SMA) is a severe, monogenetic, neuromuscular disease. A thorough understanding of its genetic cause and the availability of robust models has led to the development and approval of three gene-targeting therapies. This is a unique and exciting development for the field of neuromuscular diseases, many of which remain untreatable. The development of therapies for SMA not only opens the door to future therapeutic possibilities for other genetic neuromuscular diseases, but also informs us about the limitations of such treatments. For example, treatment response varies widely and, for many patients, significant disability remains. Currently available SMA models best recapitulate the severe types of SMA, and these models are genetically and phenotypically more homogeneous than patients. Furthermore, treating patients is leading to a shift in phenotypes with increased variability in SMA clinical presentation. Therefore, there is a need to generate model systems that better reflect these developments. Here, we will first discuss current animal models of SMA and their limitations. Next, we will discuss the characteristics required to future-proof models to assist the field in the development of additional, novel therapies for SMA." @default.
- W4387296965 created "2023-10-04" @default.
- W4387296965 creator A5011769862 @default.
- W4387296965 creator A5049948435 @default.
- W4387296965 creator A5056995428 @default.
- W4387296965 date "2023-09-01" @default.
- W4387296965 modified "2023-10-13" @default.
- W4387296965 title "Innovating spinal muscular atrophy models in the therapeutic era" @default.
- W4387296965 cites W1554872850 @default.
- W4387296965 cites W1867780591 @default.
- W4387296965 cites W1876029662 @default.
- W4387296965 cites W1969439586 @default.
- W4387296965 cites W1978885930 @default.
- W4387296965 cites W1980477780 @default.
- W4387296965 cites W1984680116 @default.
- W4387296965 cites W1986111537 @default.
- W4387296965 cites W1986399413 @default.
- W4387296965 cites W1988037421 @default.
- W4387296965 cites W1988447677 @default.
- W4387296965 cites W1992985034 @default.
- W4387296965 cites W1996670527 @default.
- W4387296965 cites W2000725203 @default.
- W4387296965 cites W2002912370 @default.
- W4387296965 cites W2013174632 @default.
- W4387296965 cites W2015026135 @default.
- W4387296965 cites W2019638822 @default.
- W4387296965 cites W2021852783 @default.
- W4387296965 cites W2033126911 @default.
- W4387296965 cites W2033608839 @default.
- W4387296965 cites W2037556712 @default.
- W4387296965 cites W2039637995 @default.
- W4387296965 cites W2045997356 @default.
- W4387296965 cites W2046790812 @default.
- W4387296965 cites W2047667011 @default.
- W4387296965 cites W2049842485 @default.
- W4387296965 cites W2054380003 @default.
- W4387296965 cites W2059920245 @default.
- W4387296965 cites W2074697255 @default.
- W4387296965 cites W2076804507 @default.
- W4387296965 cites W2077681525 @default.
- W4387296965 cites W2085465310 @default.
- W4387296965 cites W2096650232 @default.
- W4387296965 cites W2107257806 @default.
- W4387296965 cites W2109273258 @default.
- W4387296965 cites W2113007484 @default.
- W4387296965 cites W2113692867 @default.
- W4387296965 cites W2114187954 @default.
- W4387296965 cites W2115855668 @default.
- W4387296965 cites W2116175665 @default.
- W4387296965 cites W2122050746 @default.
- W4387296965 cites W2125987139 @default.
- W4387296965 cites W2131512683 @default.
- W4387296965 cites W2131951074 @default.
- W4387296965 cites W2132519155 @default.
- W4387296965 cites W2134360710 @default.
- W4387296965 cites W2134933380 @default.
- W4387296965 cites W2136868631 @default.
- W4387296965 cites W2139562720 @default.
- W4387296965 cites W2140814127 @default.
- W4387296965 cites W2140853061 @default.
- W4387296965 cites W2140859323 @default.
- W4387296965 cites W2142120640 @default.
- W4387296965 cites W2142845441 @default.
- W4387296965 cites W2147552101 @default.
- W4387296965 cites W2149237964 @default.
- W4387296965 cites W2152766938 @default.
- W4387296965 cites W2153064498 @default.
- W4387296965 cites W2153719074 @default.
- W4387296965 cites W2154357643 @default.
- W4387296965 cites W2157458455 @default.
- W4387296965 cites W2161381370 @default.
- W4387296965 cites W2165235225 @default.
- W4387296965 cites W2166723435 @default.
- W4387296965 cites W2166739815 @default.
- W4387296965 cites W2167025898 @default.
- W4387296965 cites W2167256464 @default.
- W4387296965 cites W2168115737 @default.
- W4387296965 cites W2171299886 @default.
- W4387296965 cites W2215775369 @default.
- W4387296965 cites W2225551133 @default.
- W4387296965 cites W2253617808 @default.
- W4387296965 cites W2293463841 @default.
- W4387296965 cites W2300951885 @default.
- W4387296965 cites W2344933326 @default.
- W4387296965 cites W2355313046 @default.
- W4387296965 cites W2369329430 @default.
- W4387296965 cites W2397085438 @default.
- W4387296965 cites W2473412742 @default.
- W4387296965 cites W2477368596 @default.
- W4387296965 cites W2505641731 @default.
- W4387296965 cites W2511945421 @default.
- W4387296965 cites W2530947838 @default.
- W4387296965 cites W2558044565 @default.
- W4387296965 cites W2562013491 @default.
- W4387296965 cites W2567584470 @default.
- W4387296965 cites W2568785023 @default.
- W4387296965 cites W2579663022 @default.
- W4387296965 cites W2582137641 @default.
- W4387296965 cites W2591728558 @default.
- W4387296965 cites W2606220143 @default.