المصدر: A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

المؤلفون: Children's National Research Institute

المصدر: Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

المؤلفون: California Institute for Regenerative Medicine (CIRM)

المساهمون: Tippi Mackenzie, Professor of Surgery

المصدر: A Single-Center, Non-Randomized Study of the Safety and Efficacy of in Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major
Kreger EM, Singer ST, Witt RG, Sweeters N, Lianoglou B, Lal A, Mackenzie TC, Vichinsky E. Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature. Prenat Diagn. 2016 Dec;36(13):1242-1249. doi: 10.1002/pd.4966. Epub 2016 Dec 7.
Derderian SC, Jeanty C, Walters MC, Vichinsky E, MacKenzie TC. In utero hematopoietic cell transplantation for hemoglobinopathies. Front Pharmacol. 2015 Jan 12;5:278. doi: 10.3389/fphar.2014.00278. eCollection 2014.
Vichinsky E. Advances in the treatment of alpha-thalassemia. Blood Rev. 2012 Apr;26 Suppl 1:S31-4. doi: 10.1016/S0268-960X(12)70010-3.
MacKenzie TC. Fetal Surgical conditions and the unraveling of maternal-fetal tolerance. J Pediatr Surg. 2016 Feb;51(2):197-9. doi: 10.1016/j.jpedsurg.2015.10.059. Epub 2015 Nov 4.
MacKenzie TC, David AL, Flake AW, Almeida-Porada G. Consensus statement from the first international conference for in utero stem cell transplantation and gene therapy. Front Pharmacol. 2015 Feb 10;6:15. doi: 10.3389/fphar.2015.00015. eCollection 2015. No abstract available.
Jeanty C, Derderian SC, Mackenzie TC. Maternal-fetal cellular trafficking: clinical implications and consequences. Curr Opin Pediatr. 2014 Jun;26(3):377-82. doi: 10.1097/MOP.0000000000000087.
Nijagal A, MacKenzie TC. Clinical implications of maternal-fetal cellular trafficking. Semin Pediatr Surg. 2013 Feb;22(1):62-5. doi: 10.1053/j.sempedsurg.2012.10.011.
Nijagal A, Flake AW, MacKenzie TC. In utero hematopoietic cell transplantation for the treatment of congenital anomalies. Clin Perinatol. 2012 Jun;39(2):301-10. doi: 10.1016/j.clp.2012.04.004. Epub 2012 May 8.
Nijagal A, Wegorzewska M, Le T, Tang Q, Mackenzie TC. The maternal immune response inhibits the success of in utero hematopoietic cell transplantation. Chimerism. 2011 Apr;2(2):55-7. doi: 10.4161/chim.2.2.16287.

المؤلفون: Center for Cell and Gene Therapy, Baylor College of Medicine, The Methodist Hospital Research Institute

المساهمون: Erin Morales, Assistant Professor

المصدر: T-Cell Receptor Alpha Beta+/CD19+ Depletion in Haploidentical Allogeneic Hematopoietic Cell Transplantation (allo-HCT) for Adult and Pediatric Patients with Hematological Malignancies and Non-malignant Disorders

المصدر: Maternal and Perinatal Pregnancy Outcome in Women With Heterozygous Hemoglobinopathies
Kasparek J, Burkhardt T, Hoesli I, Amstad Bencaiova G. Pregnancy outcomes in women with a hemoglobinopathy trait: a multicenter, retrospective study. Arch Gynecol Obstet. 2021 Nov;304(5):1197-1203. doi: 10.1007/s00404-021-06058-y. Epub 2021 Apr 11.

المصدر: Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders

المؤلفون: CRISPR Therapeutics

المصدر: A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated With Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

المؤلفون: CRISPR Therapeutics

المصدر: A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.
Modarai SR, Kanda S, Bloh K, Opdenaker LM, Kmiec EB. Precise and error-prone CRISPR-directed gene editing activity in human CD34+ cells varies widely among patient samples. Gene Ther. 2021 Feb;28(1-2):105-113. doi: 10.1038/s41434-020-00192-z. Epub 2020 Sep 1.

المصدر: Discarded Bone Marrow for Hematology Research

المصدر: Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies

المساهمون: Daniel Bauer, Associate Professor Pediatrics, Boston Children's Hospital

المصدر: Pilot and Feasibility Trial: Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-Hemoglobinopathies

المؤلفون: CRISPR Therapeutics

المصدر: A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Severe Sickle Cell Disease

المؤلفون: CRISPR Therapeutics

المصدر: A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia

المصدر: Long Term Follow-Up Study for Individuals With Hemoglobin Disorders After Hematopoietic Stem Cell Transplant or Gene Therapy

المؤلفون: CRISPR Therapeutics

المصدر: A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
Brusson M, Miccio A. Genome editing approaches to beta-hemoglobinopathies. Prog Mol Biol Transl Sci. 2021;182:153-183. doi: 10.1016/bs.pmbts.2021.01.025. Epub 2021 Mar 1.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. CRISPR-Cas9 Gene Editing for Sickle Cell Disease and beta-Thalassemia. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5.

المصدر: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

المؤلفون: Erlich, Henry A, Ko, Lily, Lee, Jiyae, Eaton, Katrina, Calloway, Cassandra D, Lal, Ashutosh, Das, Reena, Jamwal, Manu, Lopez-Pena, Christian, Mack, Steven J

المصدر: Croatian Medical Journal. 65(3)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Health Sciences, Pediatric, Bioengineering, Cooley's Anemia, Sickle Cell Disease, Rare Diseases, Hematology, Genetic Testing, Human Genome, Genetics, Biotechnology, Reproductive health and childbirth, Humans, Female, Pregnancy, High-Throughput Nucleotide Sequencing, Haplotypes, Polymorphism, Single Nucleotide, Prenatal Diagnosis, beta-Thalassemia, Noninvasive Prenatal Testing, beta-Globins, Genotype, Hemoglobinopathies, Anemia, Sickle Cell, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/8173f8kv
https://escholarship.org/content/qt8173f8kv/qt8173f8kv.pdf

المصدر: A Feasibility Study Evaluating Connected Bikes as a Means of Adapted Physical Activity in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Transplantation

المصدر: Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
King AA, Kamani N, Bunin N, Sahdev I, Brochstein J, Hayashi RJ, Grimley M, Abraham A, Dioguardi J, Chan KW, Douglas D, Adams R, Andreansky M, Anderson E, Gilman A, Chaudhury S, Yu L, Dalal J, Hale G, Cuvelier G, Jain A, Krajewski J, Gillio A, Kasow KA, Delgado D, Hanson E, Murray L, Shenoy S. Successful matched sibling donor marrow transplantation following reduced intensity conditioning in children with hemoglobinopathies. Am J Hematol. 2015 Dec;90(12):1093-8. doi: 10.1002/ajh.24183. Epub 2015 Oct 6.

المصدر: A Phase I/II Trial of Reduced Intensity Conditioning and Familial HLA-Mismatched Bone Marrow Transplantation in Children With Non-Malignant Disorders
Ngwube A, Shah N, Godder K, Jacobsohn D, Hulbert ML, Shenoy S. Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease. Blood Adv. 2020 Aug 25;4(16):3894-3899. doi: 10.1182/bloodadvances.2020002236.