Viral vector-based gene therapy is a transformative therapeutic approach for rare genetic diseases. The fundamental concept is to replace a transcript and/or protein that is reduced or lost due to underlying mutation(s) in the corresponding gene. Seminal examples that demonstrate the power and potential of this therapeutic strategy include AAV9-SMN1 gene therapy (trade name Zolgensma) for spinal muscular atrophy and AAV2-RPE65 (trade name Luxturna) for inherited blindness. Both treatments dramatically alter disease trajectory and provide transformative benefit for affected patients who receive it.

Ebba Andersson hade svårt att hålla sig på benen förra vintern. Dessvärre är den förmodade boven i dramat – ett smärtande knä – ett problem som vägrar ge med sig. – Det är lite oberäkneligt, säger hon inför en ny längdsäsong.

Zdobył 120 bramek dla Pogoni Szczecin, czarował na boiskach w barwach Widzewa Łódź i Arki Gdynia. Dziś Robert Dymkowski toczy nierówną walkę z nieuleczalnym stwardnieniem zanikowym bocznym. Jak dramat...

Víkend F2 v Itálii nabídl v Monze dvě vítězství Joshuy Dürksena i změny v pořadí šampionátu před finále sezóny v Madridu.

Rzadko zdarza się serial kryminalny, który potrafi jednocześnie trzymać w napięciu jak solidny thriller i boleć jak dobry dramat obyczajowy. Serial „Furious” (polski tytuł „Obsesja”) to nowa produk…

Netflix is reportedly developing a comedy series "loosely inspired" by an alleged college fraternity drug ring that will be written by and star Charlie Hall. The fictional project is being developed under writer-producer Alec Berg's overall deal with Netflix, according to Deadline. The series was rooted in allegations surrounding what authorities described as a cocaine-trafficking operation at two off-campus Penn State fraternity houses, the media outlet noted. It was not described as a dramatization of any specific case, per the outlet. Pennsylvania Attorney General Dave Sunday announced charges in August against 14 people in connection with the case, which involves multiple college students and the frat houses. Authorities said cocaine was allegedly brought from Philadelphia and New York, cut and packaged at the Delta Upsilon and Sigma Chi fraternity houses, and then distributed to Penn State students. Thirteen of those charged were current or former Penn State students at the time of the alleged conduct in 2023 and 2024, the attorney general's office said. "This was a coordinated and highly-profitable drug-trafficking crew revolving around two senior members of the fraternities, other fraternity brothers, and pledges," Sunday previously said in a news release. "In fact, according to the evidence uncovered, cutting and packaging cocaine was, for some pledges, an indoctrination into the fraternities." No title, additional cast members, production schedule or release date has been announced for Hall's Netflix project, according to Deadline. The outlet reported that Berg, a co-creator of HBO's "Barry" and an executive producer on "Silicon Valley," will executive produce and serve as supervising writer, while Amy Solomon also will executive produce through Berg's production company. Hall has appeared in "The Sex Lives of College Girls," "Bel-Air" and Apple TV's "Maximum Pleasure Guaranteed." Berg's Netflix agreement follows a lengthy run at HBO, where he also worked on "Curb Your Enthusiasm" and "Seinfeld."

Andrea Kimi Antonelli tét nélkül vág neki az Azeri Nagydíjnak. Hatalmas drámát hozott az Azeri Nagydíj pénteki időmérője, a Q1-ben Andrea Kimi Antonelli falnak csapta a Mercedest. A világbajnoki éllovas kiesett az időmérőből, hiába fért be a Q2-be így is. Jelen állás szerint a tizenhatodik helyről v

If you could start a new business right now, what would it be? View all responses I am reading Tressell’s The Ragged Trousered Philanthropists at the moment in preparation for seeing a dramat…