Today we share in Nature Reviews Genetics 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/1K4emjR…

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

Quick detection of Ab deficiency in silico: Clinical Validation of a Primary Antibody Deficiency Screening Algorithm for Primary Care pubmed.ncbi.nlm.nih.gov/37715890/

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-…

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

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

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/pr…

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.

Best way to start your week! What a refreshing manuscript! 👍🏻

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

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

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

May the 4th be with you today! 🧬

.Gaucher Community Alliance's Madelyn Schloss shares her perspective on #genetherapy at Institute for Gene Therapies & Alliance for Regenerative Medicine (ARM)'s recent #CapitolHill briefing on patient access to new innovations. View the full briefing here: gene-therapies.org/post/congressi…

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

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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 The Kohn Lab UCLA UCLA.

In a landmark scientific feat published in NEJM, a Children's Hospital & Penn Medicine 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

Allergy Immunology course October 24.

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