Pseudotyping exosomes for enhanced protein delivery in mammalian cells. [PDF]
Exosomes are cell-derived nanovesicles that hold promise as living vehicles for intracellular delivery of therapeutics to mammalian cells. This potential, however, is undermined by the lack of effective methods to load exosomes with therapeutic proteins ...
Li, Lingxuan +5 more
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Thirteen year retrospective review of the spectrum of inborn errors of metabolism presenting in a tertiary center in Saudi Arabia [PDF]
BACKGROUND: Inborn errors of metabolism (IEMs) are individually rare; however, they are collectively common. More than 600 human diseases caused by inborn errors of metabolism are now recognized, and this number is constantly increasing as new concepts ...
Abdullah Alzaben +8 more
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Mesenchymal stem cells and their use as cell replacement therapy and disease modelling tool. [PDF]
Mesenchymal stem cells (MSCs) from adult somatic tissues may differentiate in vitro and in vivo into multiple mesodermal tissues including bone, cartilage, adipose tissue, tendon, ligament or even muscle. MSCs preferentially home to damaged tissues where
García-Castro, J +5 more
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Metachromatic Leukodystrophy Clinical, Biological and Therapeutic Aspects [PDF]
Non
Abdelhedi Miled +3 more
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The clinical features and diagnosis of Metachromatic leukodystrophy: A case series of Iranian Pediatric patients [PDF]
How to Cite This Article: Jabbehdari S, Rahimian E, Jafari N, Sanii S, Khayatzadeh Kakhki S, Nejad Biglari H. The Clinical Features and Diagnosis of Metachromatic Leukodystrophy: A Case Series of Iranian Pediatric Patients. Iran J Child Neurol.
JABBEDARI, Sayena +5 more
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Remitting - Relapsing Polyneuropathy In Juvenile Metachromatic Leukodystrophy
A five-year-old girl manifested with acute relapsing polyradiculo-neuropathy. Elevated cerebrospinal fluid proteins, electro-physiological evidence of conduction block and remitting course suggested possible acquired demyelinating radiculoneuropathy ...
Taly AB +5 more
doaj
Leukodystrophy Imaging: Insights for Diagnostic Dilemmas
Leukodystrophies, a group of rare demyelinating disorders, mainly affect the CNS. Clinical presentation of different types of leukodystrophies can be nonspecific, and thus, imaging techniques like MRI can be used for a more definitive diagnosis.
Rajvi N. Thakkar +6 more
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IBMTR/ABMTR Mortimer M. Bortin Awards for Best Clinical Research Abstracts [PDF]
Mortimer M. Bortin, M.D., was one of the founding members of the International Bone Marrow Transplant Registry and served as its Scientific Director for more than 30 years. The Mortimer M. Bortin Award, established to commemorate Dr.
Gri Espinagosa, Josep +1 more
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Central Precocious Puberty in a Child With Metachromatic Leukodystrophy
Metachromatic leucodystrophy (MLD) is a rare inherited lysosomal disorder caused by reduced activity of the enzyme arylsulfatase A with accumulation of sulfatides in the nervous system.
Gilda Belli +6 more
doaj +1 more source
Late infantile metachromatic leukodystrophy: Clinical manifestations of five Taiwanese patients and Genetic features in Asia [PDF]
BACKGROUND: This study was conducted to describe the clinical and genetic features of patients with late infantile metachromatic leukodystrophy. METHODS: Clinical and genetic manifestations of five Taiwanese patients with late infantile metachromatic ...
Chi-Ren Tsai +3 more
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