Gene Therapy for Fragile X Syndrome, Challenges, and Promises
Supplying the functional protein product of the FMR1 gene to the brain is an attractive concept for curative treatment of Fragile X syndrome. Despite existing challenges, recent developments establish the basis for developing efficient gene therapy protocols for this condition.
Milen Velinov
wiley +1 more source
Intrathecal Therapies for Neurodegenerative Diseases: A Review of Current Approaches and the Urgent Need for Advanced Delivery Systems. [PDF]
Schreiner TG +3 more
europepmc +1 more source
MR‐Guidance of Gene Therapy for Brain Diseases: Moving From Palliative Treatment to Cures
ABSTRACT Regulatory bodies in the U.S. and Europe recently approved a gene therapy for aromatic L‐amino acid decarboxylase (AADC) deficiency, a rare neurologic disorder where a genetic mutation prevents dopamine production in the brain. Affected children fail to develop normal motor and cognitive functions.
Dalton H. Bermudez +2 more
wiley +1 more source
Management of High Spinal Anesthesia for Cesarean Section in the Semi-Fowler's Position. [PDF]
Martin-Orr N +4 more
europepmc +1 more source
Exertion-Induced Intrathecal Haematoma in Undiagnosed Von Willebrand Disease: A Case of Acute Spinal Cord Compression. [PDF]
Habib Z, Rasool MU, Ahmed A, Mohan R.
europepmc +1 more source
Abstract Background Impaired glymphatic clearance may contribute to pathological accumulations in Parkinson's (PD), but how it interacts with other processes causing dementia remains unclear. Diffusion tensor image analysis along the perivascular space (DTI‐ALPS) has been proposed as an indirect proxy for glymphatic clearance. Objectives To clarify DTI‐
Angeliki Zarkali +8 more
wiley +1 more source
Elevating Labor Analgesia: The Impact of Low-Dose Intrathecal Ropivacaine-Sufentanil in Combined Spinal-Epidural Analgesia: A Prospective Double-Blinded Randomized Trial. [PDF]
Ji T +6 more
europepmc +1 more source
Alcohol consumption and stroke: difficulties in assessing the relationships.
Annie Britton +8 more
core +1 more source
T2 Hyperintensities in Gracile Tracts of Cervical Spinal Cord in Giant Axonal Neuropathy (GAN)
ABSTRACT Introduction/Aims Giant axonal neuropathy (GAN) is a hereditary neurodegenerative disease due to the absence or loss of function of the gigaxonin gene. Pathologic findings in GAN are those of “dying‐back” axonal degeneration, in which the distal axon degenerates but the more proximal axon and neuronal cell body remain intact.
Diane Armao +11 more
wiley +1 more source
Optimized Kappa-Index Test Protocol to Detect Intrathecal Immunoglobulin Synthesis in Multiple Sclerosis. [PDF]
Björklund J +6 more
europepmc +1 more source

