Maria ChittyLopez retweeted
In a landmark scientific feat published in @NEJM, a @ChildrensPhila & @PennMedicine team treated the first patient with personalized #genetherapy, designed uniquely for newborn KJ, who was diagnosed with a rare #ureacycledisorder at birth. Learn more: ms.spr.ly/6016Sw9IC
>550
The 2024 IUIS Inborn Errors of Immunity (IEI) Classification has arrived! Explore our latest insights now at iuis.org/committees/iei. Find the report under “Annual 2024 Report”!
Maria ChittyLopez retweeted
I’m honored to have performed tonight, for the Paris 2024 Opening Ceremony, and so full of joy to be back in one of my very favorite cities! Most of all, I’m so happy to be celebrating these amazing athletes, with all their stories of sacrifice and determination, pain and perseverance. All of you have been so focused on your dream, and whether or not you take home a medal, I hope that being here means that it has come true for you! You should all be so proud, we know how hard you have worked to be the best of the best. Stay focused, keep going, my heart is with you! - Celine xx…
Photo: Laura Gilli
Maria ChittyLopez retweeted
.@gaucheralliance's Madelyn Schloss shares her perspective on #genetherapy at @gene_therapies & @alliancerm's recent #CapitolHill briefing on patient access to new innovations. View the full briefing here: gene-therapies.org/post/cong…
I am grateful for #CIS2024, a fantastic meeting to exchange ideas and review novel concepts.
Maria ChittyLopez retweeted
We're celebrating the International Day of Women and Girls in Science!
Let's look back on the life of Marie Skłodowska Curie: a Nobel Prize laureate who dedicated her life to science and became one of the world's greatest scientists.
#WomenInScience #NobelPrize
Maria ChittyLopez retweeted
Acid-suppressive medication and antimicrobial use in infancy associated with food allergy and anaphylaxis according to new research being presented at the 2024 AAAAI Annual Meeting. #AAAAI24 #FoodAllergy
ow.ly/MjXV50QyqAt
Best way to start your week! What a refreshing manuscript! 👍🏻
Remarkable and entertaining introduction to the field of IEI by @HsiehElena @alexbolze & Joseph Hernandez:
Inborn errors of immunity illuminate mechanisms of human immunology and pave the road to precision medicine onlinelibrary.wiley.com/doi/…
Maria ChittyLopez retweeted
Additionally, one of these therapies is the first FDA-approved treatment to utilize a type of novel genome editing technology, signaling an innovative advancement in the field of gene therapy.
Maria ChittyLopez retweeted
Today we approved two milestone treatments - the first cell-based gene therapies to treat sickle cell disease in patients 12 years and older. fda.gov/news-events/press-an…
Maria ChittyLopez retweeted
CIS's statement on the FDA's proposed rule regarding laboratory developed tests. Read the full letter here: clinimmsoc.org/.../Statement…
Maria ChittyLopez retweeted
Nobel Prize winner Katalin Karikó raised a two-time Olympic gold medalist: Here's her No. 1 piece of parenting advice cnb.cx/3F620Rt
Maria ChittyLopez retweeted
Another great manuscript from the PIDTC! Kudos to the SCID team. Post-transplant late complications increase over time. It is important to follow those patients.
jacionline.org/article/S0091…
Maria ChittyLopez retweeted
Quick detection of Ab deficiency in silico:
Clinical Validation of a Primary Antibody Deficiency Screening Algorithm for Primary Care pubmed.ncbi.nlm.nih.gov/3771…
Maria ChittyLopez retweeted
This editorial discusses the science behind a study involving 10 patients with ART-SCID, in which low-dose busulfan conditioning combined with a tweak to a gene-therapy vector resulted in favorable outcomes. nej.md/3Yzc3Hf
Maria ChittyLopez retweeted
Today we share in @NatureRevGenet a review of prime editing for precise and versatile genome manipulation. The review covers current prime editing strategies, limitations, recent developments, and applications, including work from 148 publications. 1/5
drive.google.com/file/d/1K4e…
































