Results 71 to 80 of about 8,660,824 (403)

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

open access: yesMolecular Diagnosis & Therapy, 2019
Rare diseases pose a global challenge, in that their collective impact on health systems is considerable, whereas their individually rare occurrence impedes research and development of efficient therapies.
P. Papasavva, M. Kleanthous, C. Lederer
semanticscholar   +1 more source

Genetic Relationships and Therapeutic Options for Relapsed Acute Lymphoblastic Leukemia [PDF]

open access: yes, 2020
Acute lymphoblastic leukemia (ALL) is the most common form of cancer among children and can be lethal to the adult population. Though 80% of patients with ALL reach complete remission after treatment, about 20% of those diagnosed fail to remain cancer ...
Shertzer, Hailie
core   +1 more source

Rescue of splicing-mediated intron loss maximizes expression in lentiviral vectors containing the human ubiquitin C promoter. [PDF]

open access: yes, 2014
Lentiviral vectors almost universally use heterologous internal promoters to express transgenes. One of the most commonly used promoter fragments is a 1.2-kb sequence from the human ubiquitin C (UBC) gene, encompassing the promoter, some enhancers, first
Cooper, Aaron R   +3 more
core   +1 more source

Lifestyle Behaviors and Cardiotoxic Treatment Risks in Adult Childhood Cancer Survivors

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT Background Higher doses of anthracyclines and heart‐relevant radiotherapy increase cardiovascular disease (CVD) risk. This study assessed CVD and CVD risk factors among adult childhood cancer survivors (CCSs) across cardiotoxic treatment risk groups and examined associations between lifestyle behaviors and treatment risks.
Ruijie Li   +6 more
wiley   +1 more source

Neural Excitatory/Inhibitory Imbalance in Motor Aging: From Genetic Mechanisms to Therapeutic Challenges

open access: yesBiology
Neural excitatory/inhibitory (E/I) imbalance plays a pivotal role in the aging process. However, despite its significant impact, the role of E/I imbalance in motor dysfunction and neurodegenerative diseases has not received sufficient attention.
Xuhui Chen   +6 more
doaj   +1 more source

Cerebrospinal Fluid Liquid Biopsy Enables Targeted Therapy Without Tissue Diagnosis in Pediatric Low‐Grade Gliomas With BRAF V600E Mutation

open access: yesPediatric Blood &Cancer, EarlyView.
ABSTRACT We present two pediatric cases of pediatric low‐grade gliomas (PLGG) with BRAF V600E mutations diagnosed and monitored using cerebrospinal fluid (CSF) liquid biopsy analyzed via digital droplet PCR (ddPCR), without tissue biopsy. Both patients were treated with dabrafenib and trametinib and monitored through clinical assessments, magnetic ...
Hannah Sultan   +5 more
wiley   +1 more source

Color Doppler Score: A New Approach for Monitoring a Large Placental Chorioangioma

open access: yesCase Reports in Obstetrics and Gynecology, 2014
We employed color Doppler score as an innovative approach for the prenatal diagnosis and monitoring of a large placental chorioangioma case diagnosed at 26 weeks and the subjective semiquantitative assessment of the vascularization.
Maria Angelica Zoppi   +7 more
doaj   +1 more source

Molecular and genetic alterations associated with therapy resistance and relapse of acute myeloid leukemia

open access: yesJournal of Hematology & Oncology, 2017
BackgroundThe majority of individuals with acute myeloid leukemia (AML) respond to initial chemotherapy and achieve a complete remission, yet only a minority experience long-term survival because a large proportion of patients eventually relapse with ...
H. Hackl, K. Astanina, R. Wieser
semanticscholar   +1 more source

Genetic therapy for pain management

open access: yesCurrent Review of Pain, 2000
It has been suggested that there are two main approaches to the use of gene therapy in the treatment of chronic disease, which has been confirmed by recent studies in animals.
S P, Wilson, D C, Yeomans
openaire   +3 more sources

An Overview of Leber’s Hereditary Optic Neuropathy [PDF]

open access: yes, 2015
Typically affecting males ranging from 20 to 24 years of age, Leber’s Hereditary Optic Neuropathy (LHON) is a disorder that is characterized by an acute loss of central vision.
Dalton, Matthew R
core   +2 more sources

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