Check out our recent publication, also Ellen made the cover image!: Codon-optimized Npc1 mRNA corrects Niemann-Pick type C1 disease phenotypes in vitro and in vivo: Molecular Therapy Nucleic Acids https://t.co/roScSvPovB
The September issue of Molecular Therapy Nucleic Acids is out, and the cover is ours to celebrate: Ellen Koufer’s art, Schultz lab science, work @Life4Liam helped fund.
New paper from Wohlever lab! A suite of pET28b-based Golden Gate vectors for efficient protein engineering and expression published in PROTEIN SCIENCE, lead by amazing postdoc Deepika Gaur @GaurGr https://t.co/XZwk27s0Xl. @ProteinSociety 🧵
We are thrilled to share that a week ago Jordan and I hosted our very first fundraising event for Rare Disease Day on February 28th! We poured our hearts into raising awareness and funds, spotlighting the inspiring stories of Niemann-Pick Type C families. Their courage drove us to make this event special. Grateful for everyone who joined, donated, and supported this cause close to our hearts. We were able to raise a significant amount of funds to keep our research moving forward. Together, we’re making a difference! If you want to continue to support, please donate at the link below 👇🏻
https://t.co/T38514LdRt
Stephanie Cologna explains how mass spectrometry is supporting research of Niemann-Pick Type C – and other rare degenerative #diseases 👇
https://t.co/atJIOVuHHQ
Humanized Niemann-Pick type C1 model mice illuminate potential therapies to correct protein misfolding: https://t.co/r4xnVr8JYV
Comprehensive study from Azaria, Correia, @KylieSchache et al. @M_Schultz_Lab @UMichMedSchool @uiowa
Excited to share are recent paper with the Lieberman group!
JCI Insight - Mutant induced neurons and humanized mice enable identification of Niemann-Pick C1 proteostatic therapies https://t.co/U8MEGx1E5l
Great end to the summer with our summer student Taylar doing an amazing job presenting her work on promoter strength and its impact on NPC1 trafficking at the summer undergraduate poster session!
Check out our recent collaboration with @AnnaSchwendy on NPA: Apolipoprotein-mimetic nanodiscs reduce lipid accumulation and improve... https://t.co/AxAFeQEcCG
NPC Research Update:
We're thrilled to share some amazing news from the Schultz laboratory at the University of Iowa Stead Family Children’s Hospital! In a time frame much shorter than we anticipated - only 3 months! - the team has made significant strides in our quest for an effective treatment for Niemann-Pick Type C (NPC). They have successfully packaged NPC1 mRNA into a particle, which is capable of entering cells and restoring the missing NPC1 protein. This critical breakthrough paves the way for the correction of the characteristics like cholesterol accumulation we see in NPC.
This achievement propels us into the next stages of our research - cellular dosing studies and then animal model testing. The journey is still a long way to go, but every step forward fuels our hope and resolve. These next stages will required significantly more funding, we need to raise an additional $34,000 to start the cellular dosing studies. That research will take about a year, in which time we will need to raise another $150,000 to start animal model testing of the impact of the treatment on the liver and brain. Unfortunately the research is on hold until we can fund the next stage.
Your generous support has made this possible. It's you who have empowered us and the incredible scientists at the Schultz laboratory to push the boundaries of what is possible. So, we dedicate this victory to each and every one of you, our donors and supporters. Together, we're writing a new chapter for NPC. But we still need your help to raise the funds to continue the research!
Here’s how you can continue to support Liam and help make this new treatment a reality for NPC:
Follow Liam Everywhere:
Twitter: @Life4Liam@JordanAMitchell@jlezeu
Facebook: Jordan Andrew Mitchell, Jen Lezeu
Instagram: rarekido, mitchjet, jlezeu
Share ALL of our posts about Liam and NPC:
Reposting, liking, and commenting on all of the post we make about our efforts and NPC will help spread awareness and connect with people outside our network of friends.
Donate to Liam or the Shultz Lab directly to help fund the research:
Life4Liam GoFundMe:
Our Life4Liam GoFundMe is still active while we work on setting up our nonprofit.
https://t.co/xuT7J8r3k7
Donate Directly to University of Iowa:
You can now donate directly to the University of Iowa’s Stead Family Children’s Hospital and Dr Shultz’s!
Follow the link below. Just be sure you’re donating to the “Stead Family Department of Pediatrics Development Fund” and you put “For Dr Shultz mRNA NPC Research #Life4Liam” in the “Include Gift Instructions” Section. Let us know about your donation so we can add it to our total amount raised.
https://t.co/A2II1393ru
#Life4Liam #NPC #NPCResearch #HopeForNPC #NiemannPick