Gene therapy is emerging a媽鄉s a promising candidate to t煙場reat a wide range of metabolic di物少sorders. Recently, Dr. George現玩 Mcilroy and his team at the Un民用iversity of Aberdeen Rowett Inst黑你itute and Cardiovascular a服年nd Diabetes Centre錯什 demonstrated the eff快術icacy of an Adeno-assoc美分iated Virus (AAV) gene therapy in慢學 a pre-clinical model of lipodystrophy工一 which resulted in prolong物水ed and rapid benefits t廠我o metabolic health.

Congenital generali新看zed lipodystrophy 土個type 2 (CGL) is 讀腦caused by mutations 媽西affecting the BSCL2 gen劇對e. This relatively ra路空re genetic disorder affec話離ts ~1 in a million people and cause計雨s severe metabolic disease due t很報o the critical funct木一ion of BSCL2 in a都現dipose tissue developm很校ent and maintenance. The curren朋長t therapy used to treat CGL r呢不equires daily injecti水劇ons and is not widely available.

“Our research reveals that民近 gene therapy offers great potential as工好 a treatment for lipodystrop綠外hy. Lipodystrophy is a 那妹rare disorder, where the b綠分ody cannot make or maintain adipose書是 tissue. Conseque會作ntly, individua刀舊ls with lipodystrophy can deve又綠lop severe metabolic complications, 習可including type 2 diabetes and事你 fatty liver disease,” Dr. Mcilroy said土能.

Dr.Mcilroy and his team at t市事he University of Aberd間資een were able to develop黃影 a recombinant AAV that expressed the B們還SCL2 gene and then tested their科但 therapy in pre-clin吃化ical mouse model of CGL. 空坐The team found that the家小ir therapy could successfully ta說街rget adipocyte progenitors and res一這tored expression of BSCL2 i玩樹n target tissues whi分吧ch resulted in correction of meta森術bolic phenotype門城s.

Dr. Mcilroy furth資月er elaborated, “Our findings 愛計demonstrate that a慢微deno-associated virus (AAV) mediated東離 gene therapy can r計煙estore adipose tissue develop視師ment and metabolic health in a亮這 pre-clinical mouse model o問去f lipodystrophy.”

Overall, this study demonstrates th錯呢e effectiveness of an AAV ge什區ne therapy in tre章短atment of a severe metabolic 行開disorder in a pre-clinical model 匠月and provides in妹下sight into a potential th呢匠erapeutic strategy for treatin她樂g CGL.

“We chose to source AAV vectors from Ve就錯ctorBuilder due to the e朋快ase of online vector design, provi件也sion of expert tech關姐nical support and vector production met得請hods, which ensure high titer, pur民事ity and potency,” 白不Dr. Mcilroy stated.

Construction of the AAV vectors 門林and AAV packaging was enable鐵多d by VectorBuilder’s Vector Design Studio. VectorBuilder off空道ers superior quality AAV single-s上哥tranded and self-co火山mplementary vector cloning andpackaging for your research an司事d clinical needs.

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Source: Nadine Sommer, Ahlima Roumane, 黃要Weiping Han, Mirela D謝空elibegović, Justin J. Rochford, Geor商窗ge D. Mcilroy.
Gene therapy restores adipose tissu答說e and metabolic health in a pre-clinic湖少al mouse model of lipodystrophy.
Molecular Therapy 國外- Methods & Clinical De舊快velopment, 2022
https://doi.org/10.1016/j.omtm.2022.09.雪了014