For patients with severe Leukocyte Adhesion Deficiency Type I (LAD-I), treatment options have been limited. A new gene therapy may offer hope through the FDA’s accelerated approval pathway. fda.gov/news-events/press-an…

Today, the FDA approved the first cell-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS). fda.gov/news-events/press-an…

Allergy Immunology course October 24.

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

A new study in @NEJM reports that an investigational gene therapy restored immune function in 9 children with LAD-I, a rare & life-threatening immune disorder. Those children are now living symptom-free 2 years after treatment in clinical trials co-led by @Kohn_Lab @UCLA.

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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

.@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…

May the 4th be with you today! 🧬

I am grateful for #CIS2024, a fantastic meeting to exchange ideas and review novel concepts.

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

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! 👍🏻

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.

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…

CIS's statement on the FDA's proposed rule regarding laboratory developed tests. Read the full letter here: clinimmsoc.org/.../Statement…

Nobel Prize winner Katalin Karikó raised a two-time Olympic gold medalist: Here's her No. 1 piece of parenting advice cnb.cx/3F620Rt

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…

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…

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