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Multi-Year Registry Study of Elapegademase Treatment in Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) Requiring Enzyme Replacement Therapy 多中心研究：接受酶替代疗法治疗的腺苷脱氨酶严重联合免疫缺陷（ADA-SCID）患者中elapegademase治疗的多年登记研究 Dorsey, Morna J.; Butte, Manish J.; Lieberman, Jay A.; Lehman, Heather; Fausnight, Tracy; Keller, Michael D.; Fradette, Caroline; Hershfield, Michael S.; Pozos, Tamara C.; Rozova, Anna; Wall, Luke A.; Bednarski, Jeffrey J.; Tarrant, Teresa K.; Chong, Hey J.; Geng, Bob; Temin, Noemi Toiber; Laubach, Susan S.; Lin, Leo; Mousallem, Talal; Walter, Jolan E. 分享 收藏
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Neurologic Status of Patients with Purine Nucleoside Phosphorylase Deficiency Before and After Hematopoetic Stem Cell Transplantation Karaaslan, Betul Gemici; Turan, Isilay; Aydemir, Sezin; Meric, Zeynep Akyuncu; Atay, Didem; Akcay, Arzu; Sari, Aysun Ayaz; Hershfield, Michael; Cipe, Funda; Aksoy, Basak Adakli; Ersoy, Gizem Zengin; Bozkurt, Ceyhun; Demirkol, Yasemin Kendir; Ozturk, Gulyuz; Aydogmus, Cigdem; Kiykim, Ayca; Cokugras, Haluk 分享 收藏
Comparison of disease phenotypes and mechanistic insight on causal variants in patients with DADA2 Chen, Liang; Mamutova, Anna; Kozlova, Anna; Latysheva, Elena; Evgeny, Frolov; Latysheva, Tatiana; Savostyanov, Kirill; Pushkov, Alexander; Zhanin, Ilya; Raykina, Elena; Kurnikova, Maria; Mersiyanova, Irina; Platt, Craig D.; Jee, Hyuk; Brodeur, Kailey; Du, Yan; Liu, Meng; Weiss, Aaron; Schulert, Grant S.; Rodriguez-Smith, Jackeline; Hershfield, Michael S.; Aksentijevich, Ivona; Zhou, Qing; Nigrovic, Peter A.; Shcherbina, Anna; Alexeeva, Ekaterina; Lee, Pui Y. 分享 收藏
Long-Term Immune Reconstitution in ADA-Deficient Patients Treated With Elapegademase: A Real-World Experience Murguia-Favela, Luis; Suresh, Sneha; Wright, Nicola A. M.; Alvi, Saima; Tehseen, Sarah; Hernandez-Trujillo, Vivian; Seroogy, Christine M.; Haddad, Elie; Nieves, Daime; Hershfield, Michael S.; Walter, Jolan E.; Pettiford, Leah; Kamani, Naynesh R.; Keller, Michael D.; Pham-Huy, Anne; Grunebaum, Eyal 分享 收藏
Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency Barzaghi, Federica; Cicalese, Maria Pia; Zoccolillo, Matteo; Brigida, Immacolata; Barcella, Matteo; Merelli, Ivan; Sartirana, Claudia; Zanussi, Monica; Calbi, Valeria; Bernardo, Maria Ester; Tucci, Francesca; Migliavacca, Maddalena; Giglio, Fabio; Doglio, Matteo; Canarutto, Daniele; Ferrua, Francesca; Consiglieri, Giulia; Prunotto, Giulia; Saettini, Francesco; Bonanomi, Sonia; Rovere-Querini, Patrizia; Di Colo, Giulia; Jofra, Tatiana; Fousteri, Georgia; Penco, Federica; Gattorno, Marco; Hershfield, Michael S.; Bongiovanni, Lucia; Ponzoni, Maurilio; Marktel, Sarah; Milani, Raffaella; Peccatori, Jacopo; Ciceri, Fabio; Mortellaro, Alessandra; Aiuti, Alessandro 分享 收藏
Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients (vol 41, pg 1633, 2021) Hashem, Hasan; Bucciol, Giorgia; Ozen, Seza; Unal, Sule; Bozkaya, Ikbal Ok; Akarsu, Nurten; Taskinen, Mervi; Koskenvuo, Minna; Saarela, Janna; Dimitrova, Dimana; Hickstein, Dennis D.; Hsu, Amy P.; Holland, Steven M.; Krance, Robert; Sasa, Ghadir; Kumar, Ashish R.; Mueller, Ingo; de Sousa, Monica Abreu; Delafontaine, Selket; Moens, Leen; Babor, Florian; Barzaghi, Federica; Cicalese, Maria Pia; Bredius, Robbert; van Montfrans, Joris; Baretta, Valentina; Cesaro, Simone; Stepensky, Polina; Benedicte, Neven; Moshous, Despina; Le Guenno, Guillaume; Boutboul, David; Dalal, Jignesh; Brooks, Joel P.; Dokmeci, Elif; Dara, Jasmeen; Lucas, Carrie L.; Hambleton, Sophie; Wilson, Keith; Jolles, Stephen; Koc, Yener; Gungor, Tayfun; Schnider, Caroline; Candotti, Fabio; Steinmann, Sandra; Schulz, Ansgar; Chambers, Chip; Hershfield, Michael; Ombrello, Amanda; Kanakry, Jennifer A.; Meyts, Isabelle 分享 收藏
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Polyethylene Glycol-Like Brush Polymer Conjugate of a Protein Drug Does Not Induce an Antipolymer Immune Response and Has Enhanced Pharmacokinetics than Its Polyethylene Glycol Counterpart 蛋白质药物的聚乙二醇样刷状聚合物缀合物不会诱导抗聚合物免疫反应，并且比其聚乙二醇对应物具有增强的药代动力学 Ozer, Imran; Kelly, Garrett; Gu, Renpeng; Li, Xinghai; Zakharov, Nikita; Sirohi, Parul; Nair, Smita K.; Collier, Joel H.; Hershfield, Michael S.; Hucknall, Angus M.; Chilkoti, Ashutosh 分享 收藏
Comprehensive analysis of ADA2 genetic variants and estimation of carrier frequency driven by a function-based approach Jee, Hyuk; Huang, Zhengping; Baxter, Samantha; Huang, Yuelong; Taylor, Maria L.; Henderson, Lauren A.; Rosenzweig, Sofia; Sharma, Aman; Chambers, Eugene P.; Hershfield, Michael S.; Zhou, Qing; Dedeoglu, Fatma; Aksentijevich, Ivona; Nigrovic, Peter A.; O'Donnell-Luria, Anne; Lee, Pui Y. 分享 收藏
Intrinsic Defects in B Cell Development and Differentiation, T Cell Exhaustion and Altered Unconventional T Cell Generation Characterize Human Adenosine Deaminase Type 2 Deficiency B细胞发育和分化的内在缺陷，T细胞耗竭和非常规T细胞生成的改变是人类腺苷脱氨酶2型缺乏症的特征 Yap, Jin Yan; Moens, Leen; Lin, Ming-Wei; Kane, Alisa; Kelleher, Anthony; Toong, Catherine; Wu, Kathy H. C.; Sewell, William A.; Phan, Tri Giang; Hollway, Georgina E.; Enthoven, Karen; Gray, Paul E.; Casas-Martin, Jose; Wouters, Carine; De Somer, Lien; Hershfield, Michael; Bucciol, Giorgia; Delafontaine, Selket; Ma, Cindy S.; Tangye, Stuart G.; Meyts, Isabelle 分享 收藏
Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients 造血干细胞移植治疗腺苷脱氨酶2缺乏症30例报告 Hashem, Hasan; Bucciol, Giorgia; Ozen, Seza; Unal, Sule; Bozkaya, Ikbal Ok; Akarsu, Nurten; Taskinen, Mervi; Koskenvuo, Minna; Saarela, Janna; Dimitrova, Dimana; Hickstein, Dennis D.; Hsu, Amy P.; Holland, Steven M.; Krance, Robert; Sasa, Ghadir; Kumar, Ashish R.; Muller, Ingo; de Sousa, Monica Abreu; Delafontaine, Selket; Moens, Leen; Babor, Florian; Barzaghi, Federica; Cicalese, Maria Pia; Bredius, Robbert; van Montfrans, Joris; Baretta, Valentina; Cesaro, Simone; Stepensky, Polina; Benedicte, Neven; Moshous, Despina; Le Guenno, Guillaume; Boutboul, David; Dalal, Jignesh; Brooks, Joel P.; Dokmeci, Elif; Dara, Jasmeen; Lucas, Carrie L.; Hambleton, Sophie; Wilson, Keith; Jolles, Stephen; Koc, Yener; Gungor, Tayfun; Schnider, Caroline; Candotti, Fabio; Steinmann, Sandra; Schulz, Ansgar; Chambers, Chip; Hershfield, Michael; Ombrello, Amanda; Kanakry, Jennifer A.; Meyts, Isabelle 分享 收藏
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Normal IgH Repertoire Diversity in an Infant with ADA Deficiency After Gene Therapy Baloh, Carolyn H.; Borkar, Samiksha A.; Chang, Kai-Fen; Yao, Jiqiang; Hershfield, Michael S.; Parikh, Suhag H.; Kohn, Donald B.; Goodenow, Maureen M.; Sleasman, John W.; Yin, Li 分享 收藏
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency 自体慢病毒基因治疗腺苷脱氨酶缺乏症 Kohn, D. B.; Booth, C.; Shaw, K. L.; Xu-Bayford, J.; Garabedian, E.; Trevisan, V.; Carbonaro-Sarracino, D. A.; Soni, K.; Terrazas, D.; Snell, K.; Ikeda, A.; Leon-Rico, D.; Moore, T. B.; Buckland, K. F.; Shah, A. J.; Gilmour, K. C.; De Oliveira, S.; Rivat, C.; Crooks, G. M.; Izotova, N.; Tse, J.; Adams, S.; Shupien, S.; Ricketts, H.; Davila, A.; Uzowuru, C.; Icreverzi, A.; Barman, P.; Fernandez, B. Campo; Hollis, R. P.; Coronel, M.; Yu, A.; Chun, K. M.; Casas, C. E.; Zhang, R.; Arduini, S.; Lynn, F.; Kudari, M.; Spezzi, A.; Zahn, M.; Heimke, R.; Labik, I.; Parrott, R.; Buckley, R. H.; Reeves, L.; Cornetta, K.; Sokolic, R.; Hershfield, M.; Schmidt, M.; Candotti, F.; Malech, H. L.; Thrasher, A. J.; Gaspar, H. B. 分享 收藏
Spectrum of Systemic Auto-Inflammatory Diseases in India: A Multi-Centric Experience Suri, Deepti; Rawat, Amit; Jindal, Ankur Kumar; Vignesh, Pandiarajan; Gupta, Anju; Pilania, Rakesh Kumar; Joshi, Vibhu; Arora, Kanika; Kumrah, Rajni; Anjani, Gummadi; Aggarwal, Amita; Phadke, Shubha; Aboobacker, Fouzia N.; George, Biju; Edison, Eunice Sindhuvi; Desai, Mukesh; Taur, Prasad; Gowri, Vijaya; Pandrowala, Ambreen Abdulwahab; Bhattad, Sagar; Kanakia, Swati; Gottorno, Marco; Ceccherini, Isabella; Almeida de Jesus, Adriana; Goldbach-Mansky, Raphaela; Hershfield, Michael S.; Singh, Surjit 分享 收藏
Clinical, Immunological, and Molecular Features of Severe Combined Immune Deficiency: A Multi-Institutional Experience From India Vignesh, Pandiarajan; Rawat, Amit; Kumrah, Rajni; Singh, Ankita; Gummadi, Anjani; Sharma, Madhubala; Kaur, Anit; Nameirakpam, Johnson; Jindal, Ankur; Suri, Deepti; Gupta, Anju; Khadwal, Alka; Saikia, Biman; Minz, Ranjana Walker; Sharma, Kaushal; Desai, Mukesh; Taur, Prasad; Gowri, Vijaya; Pandrowala, Ambreen; Dalvi, Aparna; Jodhawat, Neha; Kambli, Priyanka; Madkaikar, Manisha Rajan; Bhattad, Sagar; Ramprakash, Stalin; Raghuram, C. P.; Jayaram, Ananthvikas; Sivasankaran, Meena; Munirathnam, Deenadayalan; Balaji, Sarath; Rajendran, Aruna; Aggarwal, Amita; Singh, Komal; Na, Fouzia; George, Biju; Mehta, Ankit; Lashkari, Harsha Prasada; Uppuluri, Ramya; Raj, Revathi; Bartakke, Sandip; Gupta, Kirti; Sreedharanunni, Sreejesh; Ogura, Yumi; Kato, Tamaki; Imai, Kohsuke; Chan, Koon Wing; Leung, Daniel; Ohara, Osamu; Nonoyama, Shigeaki; Hershfield, Michael; Lau, Yu-Lung; Singh, Surjit 分享 收藏
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