Unraveling the Genetic Landscape of Neurological Disorders: Insights into Pathogenesis, Techniques for Variant Identification, and Therapeutic Approaches
Z Firdaus, X Li - International journal of molecular sciences, 2024 - mdpi.com
Genetic abnormalities play a crucial role in the development of neurodegenerative disorders
(NDDs). Genetic exploration has indeed contributed to unraveling the molecular …
(NDDs). Genetic exploration has indeed contributed to unraveling the molecular …
Survival After Hematopoietic Stem Cell Transplantation in Severe Combined Immunodeficiency (SCID): A Worldwide Review of the Prognostic Variables
GA Goebel, CS de Assis, LAO Cunha… - Clinical Reviews in …, 2024 - Springer
This study aims to perform an extensive review of the literature that evaluates various factors
that affect the survival rates of patients with severe combined immunodeficiency (SCID) after …
that affect the survival rates of patients with severe combined immunodeficiency (SCID) after …
[HTML][HTML] Secondary bone marrow graft loss after third-party virus-specific T cell infusion: Case report of a rare complication
MD Keller, SA Schattgen, S Chandrakasan… - Nature …, 2024 - nature.com
Virus-specific T cells (VST) from partially-HLA matched donors have been effective for
treatment of refractory viral infections in immunocompromised patients in prior studies with a …
treatment of refractory viral infections in immunocompromised patients in prior studies with a …
Lung cell transplantation for pulmonary fibrosis
I Milman Krentsis, Y Zheng, C Rosen, SY Shin… - Science …, 2024 - science.org
Idiopathic pulmonary fibrosis is a major cause of death with few treatment options. Here, we
demonstrate the therapeutic efficacy for lung fibrosis of adult lung cell transplantation using …
demonstrate the therapeutic efficacy for lung fibrosis of adult lung cell transplantation using …
[HTML][HTML] Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II …
Viral infections remain a major risk in immunocompromised pediatric patients, and virus-
specific T cell (VST) therapy has been successful for treatment of refractory viral infections in …
specific T cell (VST) therapy has been successful for treatment of refractory viral infections in …
Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency
Recombination-activating genes (RAG1 and RAG2) are critical for lymphoid cell
development and function by initiating the variable (V), diversity (D), and joining (J)(V (D) J) …
development and function by initiating the variable (V), diversity (D), and joining (J)(V (D) J) …
Synergistic effect and molecular mechanism of PVA and UM171 in ex vivo expansion of primitive hematopoietic stem cells
Y Ren, Y Cui, J Feng, Y Tan, F Ren… - Journal of Cellular …, 2024 - Wiley Online Library
Umbilical cord blood (UCB) is a valuable source of hematopoietic stem cells (HSCs) used
for transplantation; the number of cells in a single UCB is too small to quickly establish bone …
for transplantation; the number of cells in a single UCB is too small to quickly establish bone …
Infectious complications in the pediatric immunocompromised host: a narrative review
T Lehrnbecher, AH Groll - Clinical Microbiology and Infection, 2024 - Elsevier
Background Infections are a major cause for morbidity in children with primary or secondary
immunodeficiency, and have a negative impact on overall outcome. Objectives This …
immunodeficiency, and have a negative impact on overall outcome. Objectives This …
JAK/STAT defects and immune dysregulation, and guiding therapeutic choices
NS Chaimowitz, MR Smith… - Immunological …, 2024 - Wiley Online Library
Inborn errors of immunity (IEIs) encompass a diverse spectrum of genetic disorders that
disrupt the intricate mechanisms of the immune system, leading to a variety of clinical …
disrupt the intricate mechanisms of the immune system, leading to a variety of clinical …
[HTML][HTML] Quality considerations and major pitfalls for high throughput DNA-based newborn screening for severe combined immunodeficiency and spinal muscular …
J Bzdok, L Czibere, S Burggraf, O Landt, EM Maier… - Plos one, 2024 - journals.plos.org
Background Many newborn screening programs worldwide have introduced screening for
diseases using DNA extracted from dried blood spots (DBS). In Germany, DNA-based …
diseases using DNA extracted from dried blood spots (DBS). In Germany, DNA-based …