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Journal Articles

Systematic functional validation of IKAROS variants from patients and laboratory-generated mutations

Open Access
Hye Sun Kuehn, Agustin Alejandro Gil Silva, Natchanun Klangkalya, Ana Esteve-Sole, Shubham Goel, Julie E Niemela, Jennifer L Stoddard, Hey Chong, Kelli W Williams, Winnie Ip, Mario Framil Seoane, Sathish K Loganathan, Ariharan Anantharachagan, Ana García-Soidán, Serena Beth Shaffren, Constance Bindernagel, Philip Mendez, Rahim Z Miller, Hassan Abolhassani, David P Hoytema van Konijnenburg, Ashley V Geerlinks, Diana Cabanillas, Evelien Zonneveld-Huijssoon, Abraham Rutgers, Stuart G. Tangye, Cindy S Ma, Alice Grey, Patrick O'Young, Juan Carlos Aldave Becerra, Jonathan J Lyons, Andrew White, Michael Claiborne, Ali Doroudchi, Brian D Modena, Joshua D. Milner, Xavier Solanich, João Farela Neves, Craig Platt, Lennart Hammarström, John Routes, James Verbsky, Manish Butte, Harry Lesmana, William JH Griffiths, Dinakantha S Kumararatne, Fabian Hauck, Luis M Allende, Carlos Rodríguez-Gallego, Reem Elfeky, Tamar Sarah Rubin, Kaan Boztug, Thomas A. Fleisher, Sergio D Rosenzweig
Journal: Blood Advances
Blood Adv bloodadvances.2025017243.
https://doi.org/10.1182/bloodadvances.2025017243
Published: 2025
Includes: Supplemental data
Journal Articles

Hematopoietic Cell Transplantation for Wiskott-Aldrich syndrome: A PIDTC Report

Open Access
Jessie L Alexander, Blachy J. Dávila Saldaña, Ruta Brazauskas, Sravya Gethika Dammalapati, Linda M Griffith, Ami J. Shah, Kristin A Shimano, Hans D Ochs, Jack Bleesing, Christen L Ebens, Malika Kapadia, Andrea Bauchat, Neena Kapoor, Joseph H. Oved, Hesham Eissa, Hannah Lust, Michael D. Keller, Hilary Haines, Shanmuganathan Chandrakasan, Julie-An M. Talano, Ahmad Rayes, Lisa M. Madden, Evan B. Shereck, Holly K Miller, Lisa R Forbes Satter, Caridad A. Martinez, Jacob Rozmus, Jeffrey J Bednarski, Lolie C Yu, Deepakbabu Chellapandian, Victor M. Aquino, Alan P Knutsen, Hey Chong, Ashley Chopek, Alfred P Gillio, Avni Joshi, Hemalatha G. Rangarajan, Theodore B Moore, Jeffrey R. Andolina, Kenneth DeSantes, Mark T Vander Lugt, Susan E Prockop, David Shyr, Kathleen E. Sullivan, Suhag H Parikh, Katja G. Weinacht, Troy R. Torgerson, Rebecca A Marsh, Christopher C Dvorak, Alice Y Chan, Elie Haddad, Jennifer Heimall, Michael A. Pulsipher, Jennifer W Leiding, Donald B. Kohn, Jennifer M Puck, Luigi D Notarangelo, David J Rawlings, Morton J Cowan, Aleksandra Petrovic, Sung-Yun Pai, Lauri M. Burroughs
Journal: Blood Advances
Blood Adv bloodadvances.2025017662.
https://doi.org/10.1182/bloodadvances.2025017662
Published: 2025
Includes: Supplemental data
Journal Articles

Long-term treatment with selective PI3Kδ inhibitor leniolisib in adults with activated PI3Kδ syndrome

Free
Clinical Trials & Observations
V. Koneti Rao, Elaine Kulm, Jennifer Grossman, David Buchbinder, Hey Chong, Jason Bradt, Sharon Webster, Anna Šedivá, Virgil A. Dalm, Gulbu Uzel
Journal: Blood Advances
Blood Adv (2024) 8 (12): 3092–3108.
https://doi.org/10.1182/bloodadvances.2023011000
Published: 2024
Includes: Supplemental data