Last Posted: Aug 19, 2021
- Spectrum of Hematological Malignancies, Clonal Evolution and Outcomes in 144 Mayo Clinic Patients with Germline Predisposition Syndromes.
St Martin Emma et al. American journal of hematology 2021
- Cell and Gene Therapy for Anemia: Hematopoietic Stem Cells and Gene Editing.
Anurogo Dito et al. International journal of molecular sciences 2021 22(12)
- Monitoring and treatment of MDS in genetically susceptible persons.
Davies Stella M et al. Hematology. American Society of Hematology. Education Program 2019 2019(1) 105-109
- [Challenges of screening germline predispositions in children].
Manabe Atsushi et al. [Rinsho ketsueki] The Japanese journal of clinical hematology 2020 61(6) 682-686
- Grants for Rare Disease Research
F Sankar, JAMA< November 19, 2019
- Could editing the DNA of embryos with CRISPR help save people who are already alive?
A Joseph, StatNews, September 16, 2019
- Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia
P Rio et al, Nature Medicine, September 9, 2019
- Current Knowledge and Priorities for Future Research in Late Effects after Hematopoietic Cell Transplantation for Inherited Bone Marrow Failure Syndromes: Consensus Statement from the Second Pediatric Blood and Marrow Transplant Consortium International Conference on Late Effects after Pediatric Hematopoietic Cell Transplantation.
Dietz Andrew C et al. Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation 2017 May 23(5) 726-735
- PARP Inhibitors Hit the Pancreatic Cancer Scene
D Ternyila, Oncology Nursing News, August 9, 2019
- [Genetic analysis of Japanese patients with Fanconi anemia: novel findings].
Mori Minako et al. [Rinsho ketsueki] The Japanese journal of clinical hematology 2019 60(6) 691-701
Rare Disease PHGKB is an online, continuously updated, searchable database of published scientific literature, CDC and NIH resources, and other information that address the public health impact and translation of genomic and other precision health discoveries into improved health outcomes related to rare diseases...more
Selected Rare Diseases
- Alpha-1 Antitrypsin Deficiency
- Amyotrophic Lateral Sclerosis
- Brugada Syndrome
- Cerebral Palsy
- Cystic Fibrosis
- Duchenne Muscular Dystrophy
- Erythema Multiforme
- Familial Mediterranean Fever
- Fragile X Syndrome
- Gaucher Disease
- Graves Disease
- Huntington Disease
- Myasthenia Gravis
- Retinitis Pigmentosa
- Severe Combined Immunodeficiency
Disclaimer: Articles listed in the Public Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.