Intrathecal (IT) injection of adeno-associated virus (AAV) has drawn considerable interest in CNS gene therapy by virtue of its safety, noninvasiveness, and excellent transduction efficacy in the CNS. Previous studies have demonstrated the therapeutic potency of AAV-delivered gene therapy in neurodegenerative disorders by IT administration. However, high rates of unpredictable failure due to the technical limitation of IT administration in small animals have been reported. Here, we established a scoring system to indicate the success extent of lumbar puncture in small animals by adding 1% lidocaine hydrochloride into the injection solution. We further show that the extent of transient weakness following injection can predict the transduction efficiency of AAV. Thus, this IT injection method can be used to optimize therapeutic trials in mouse models of CNS diseases that afflict wide regions of the CNS.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.3791/58565 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Therapeutic potential of siRNA PMP22-SQ nanoparticles for Charcot-Marie-Tooth 1A neuropathy in rodents and non-human primates.
Int J Pharm
January 2025
Université Paris-Saclay, Inserm, Maladies et hormones du système nerveux, 94276 Le Kremlin-Bicêtre, France. Electronic address:
Small interfering RNA (siRNA) has shown promising results for the treatment of Charcot-Marie-Tooth disease 1A (CMT1A) caused by overexpression of peripheral myelin protein (PMP22), leading to myelin dysfunction and axonal damage. Recently, we developed siRNA PMP22-squalene (SQ) nanoparticles (NPs) for intravenous use. Three consecutive injections of siRNA PMP22-SQ NPs at a cumulative dose of 1.
View Article and Find Full Text PDFShoulder Aesthetics: A Novel Synergy of Hyaluronic Acid Fillers and Neuromodulators.
Aesthetic Plast Surg
January 2025
Medical Research Inc., Wonju, Korea.
The pursuit of aesthetically pleasing shoulder contours, particularly those characterised by a sharp, angular definition, has gained significant traction in Southeast Asia. Traditionally, neuromodulators have been used to achieve these results by inducing muscle atrophy, particularly in the trapezius muscles. However, this approach can carry potential risks, such as compromised muscle function and spinal instability.
View Article and Find Full Text PDFDisappearance of spinal catheter tip due to catheter stretching: a case report.
Reg Anesth Pain Med
January 2025
Anaesthesia, University Hospital Waterford, Waterford, Ireland.
Background: Continuous spinal anesthesia (CSA) offers precise, prolonged neuraxial anesthesia suitable for high-risk patients. This technique minimizes hemodynamic instability but comes with notable challenges. Vigilant catheter management is crucial to avoid complications, including the risk of catheter retention.
View Article and Find Full Text PDFProne Transpsoas Lumbar Interbody Fusion for Degenerative Disc Disease.
JBJS Essent Surg Tech
January 2025
Department of Neurosurgery, Center for Neuroscience and Spine, Virginia Mason Medical Center, Seattle, Washington.
Background: Prone transpsoas lumbar interbody fusion (PTP) is a newer technique to treat various spinal disc pathologies. PTP is a variation of lateral lumbar interbody fusion (LLIF) that is performed with the patient prone rather than in the lateral decubitus position. This approach offers similar benefits of lateral spinal surgery, which include less blood loss, shorter hospital stay, and quicker recovery compared with traditional open spine surgery.
View Article and Find Full Text PDFPossible involvement of vitamin K insufficiency in the progression of cervical ossification of the posterior longitudinal ligament.
Sci Rep
January 2025
Department of Orthopaedics, Jichi Medical University School of Medicine, Tochigi, 329-0498, Japan.
Ossification of the posterior longitudinal ligament of the vertebral column (OPLL) is a disease characterised by ectopic bone formation in the spinal ligament that causes progressive neurological impairment. However, there are no suitable treatments for OPLL. Here, we compared the general characteristics and haemostasis of patients with OPLL and those with cervical spondylotic myelopathy.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!