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Literature DB >> 27419862

Gene Therapy for "Bubble Boy" Disease.

Abstract

Adenosine deaminase (ADA) deficiency results in the accumulation of toxic metabolites that destroy the immune system, causing severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease. Strimvelis is a European Medicines Agency approved gene therapy for ADA-SCID patients without a suitable bone marrow donor.

Entities: Disease Gene Species

Year: 2016 PMID： 27419862 DOI： 10.1016/j.cell.2016.06.049

Source DB: PubMed Journal: Cell ISSN： 0092-8674 Impact factor: 41.582

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Cited

12 in total

1. Metabolic engineering generates a transgene-free safety switch for cell therapy.

Authors: Volker Wiebking; James O Patterson; Renata Martin; Monica K Chanda; Ciaran M Lee; Waracharee Srifa; Gang Bao; Matthew H Porteus
Journal: Nat Biotechnol Date: 2020-07-13 Impact factor: 54.908

Review 2. The delivery challenge: fulfilling the promise of therapeutic genome editing.

Authors: Joost van Haasteren; Jie Li; Olivia J Scheideler; Niren Murthy; David V Schaffer
Journal: Nat Biotechnol Date: 2020-06-29 Impact factor: 54.908

3. Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.

Authors: Sara El Ashkar; Dominique Van Looveren; Franziska Schenk; Lenard S Vranckx; Jonas Demeulemeester; Jan De Rijck; Zeger Debyser; Ute Modlich; Rik Gijsbers
Journal: Mol Ther Nucleic Acids Date: 2017-04-12

Review 4. Bringing Neural Cell Therapies to the Clinic: Past and Future Strategies.

Authors: Stefan Irion; Susan E Zabierowski; Mark J Tomishima
Journal: Mol Ther Methods Clin Dev Date: 2016-12-24 Impact factor: 6.698

5. The Pharmacology of Gene and Cell Therapy.

Authors: Thomas J Conlon; Fulvio Mavilio
Journal: Mol Ther Methods Clin Dev Date: 2018-03-15 Impact factor: 6.698

6. Tailored Cytokine Optimization for ex vivo Culture Platforms Targeting the Expansion of Human Hematopoietic Stem/Progenitor Cells.

Authors: André Branco; Sara Bucar; Jorge Moura-Sampaio; Carla Lilaia; Joaquim M S Cabral; Ana Fernandes-Platzgummer; Cláudia Lobato da Silva
Journal: Front Bioeng Biotechnol Date: 2020-09-25

Gene Therapy for "Bubble Boy" Disease.

1. Metabolic engineering generates a transgene-free safety switch for cell therapy.

Review 2. The delivery challenge: fulfilling the promise of therapeutic genome editing.

3. Engineering Next-Generation BET-Independent MLV Vectors for Safer Gene Therapy.

Review 4. Bringing Neural Cell Therapies to the Clinic: Past and Future Strategies.

5. The Pharmacology of Gene and Cell Therapy.

6. Tailored Cytokine Optimization for ex vivo Culture Platforms Targeting the Expansion of Human Hematopoietic Stem/Progenitor Cells.

Review 7. Engineering adeno-associated virus vectors for gene therapy.

Review 8. Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.

Review 9. Gene Therapy With Regulatory T Cells: A Beneficial Alliance.

Review 10. An overview of development in gene therapeutics in China.