Publications | Sickle Cell Center for Excellence

Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.

Transplantation and cellular therapy

Sharma A, Kassim A, Thompson A, Williams DA, Liu HD, Boelens JJ, LaBelle JL, Tisdale J, de la Fuente J, Kanter J, Krishnamurti L, Pecker LH, Walters MC, Eapen M, Porteus M, Bhatia M, Soni S, Corbacioglu S, John TD, Cancio MI
Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia.

Blood

Kwiatkowski JL, Thompson AA, Schneiderman J, Thuret I, Kulozik AE, Yannaki E, Cavazzana M, Hongeng S, Olson TS, Sauer MG, Thrasher AJ, Lal A, Rasko JE, Kunz JB, Kinney MA, Chawla A, Ali S, Tao G, Thakar H, Paramore C, Witthuhn N, Walters MC, Locatelli F
Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease.

The New England journal of medicine

Frangoul H, de la Fuente J, Chopra Y, Meisel R, Amrolia PJ, Algeri M, Sharma A, Cappellini MD, Corbacioglu S, Kattamis A, Lobitz S, de Montalembert M, Rondelli D, Sheth S, Steinberg MH, Walters MC, Boerner K, Liu T, Zairis S, Hobbs W, Grupp SA, Locatelli F, CLIMB THAL-141 and CLIMB SCD-151 Study Groups
Epidemiology of Pediatric Transfusion Reactions.

JAMA network open

Stone EF, Chacreton D, Jimenez A, Kelly S, Goel R, Hendrickson JE, Nellis ME, Sola-Visner M, Karam O, Patel RM, Vossoughi S, Vissa M, Wilson S, VanWormer JJ, Singer ST, Bakhtary S, Vichinsky EP, Luban NLC, Josephson CD, Karafin MS, NHLBI Recipient Epidemiology and Donor Evaluation Study-IV-Pediatric (REDS-IV-P)
Sustainment of Hydroxyurea Adherence in Patients With Sickle Cell Disease.

JAMA network open

Heitzer AM, Wooten Z, Luo G, Treadwell M, King AA, Gordeuk VR, Shah N, Abrams CM, McCuskee S, Gollan S, Longoria J, Hankins JS, Sickle Cell Disease Implementation Consortium
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia.

The New England journal of medicine

Frangoul H, Hanna R, Walters MC, Kao RL, Carroll C, McManus M, Chang KH, Jaskolka MC, Kim K, Yu Q, Badamosi N, Mei B, Afonja O, Thompson A, EdiThal Investigators
In Utero Hematopoietic Cell Transplantation in Fetuses with Alpha Thalassemia Major: A Phase 1 Clinical Trial.

Blood advances

MacKenzie TC, Lianoglou BR, Gonzalez J, Dvorak CC, Kharbanda S, Herzeg A, Canepa E, Kreger EM, Frascoli M, Sper R, Gupta A, Leung J, Tang Q, Sinha R, Worthington AK, Gano D, Coco Bakkour S, Chafets D, Keller RL, Shimotake J, Lal A, Glenn OA, Moon-Grady A, Vichinsky EP
Cure Sickle Cell Initiative Recommendations on Common Data Elements for Sickle Cell Disease Gene Therapy Trials.

Blood advances

Lanzkron S, Coleman-Cowger VH, Thompson AA, Carroll CP, Clemons T, DeBaun MR, Washko Kanter J, Malik P, Manwani DG, Pierciey FJ, Walters MC, Alai S
Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.

bioRxiv : the preprint server for biology

Jia K, Soupene E, Sinha R, Lesch BJ, Pendergast MA, Choi R, Zhang X, Foppiani EM, Kostamo Z, Chu SN, Sharma D, Yu X, Cordero M, Walters MC, MacKenzie TC, Sheehan VA, Cromer MK
A blueprint to accelerate rare pediatric gene therapy approvals.

Nature medicine

Abou-El-Enein M, Walters MC, Sanghavi D, Kohn DB
CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease.

The New England journal of medicine

Hanna R, Frangoul H, Pineiro L, McKinney C, Mapara M, Dalal J, Rangarajan HG, Atkins H, Sharma A, Chang KH, Jaskolka MC, Kim K, Yu Q, Mei B, Afonja O, Walters MC, RUBY Investigators
Futuristic global cell and gene therapy regulations based on a review of today's leading frameworks.

Cytotherapy

Hashmi SK, Jamil MA, de la Fuente J, Mahlangu J, Ozelo MC, Walters MC, Almannaei A, Srivastava A, Alzahrani H, Lu P, Kenet G, Nomura K, Leboulch P