One of the greatest barriers for the use of adeno-associated vectors (AAV) in gene therapy is the presence of pre-existing antibodies against AAV in the general population. Since many of the anti-AAV antibodies have the ability to neutralize the transduction target tissues, even patients with low antibody titers must be excluded from clinical trials or therapy. In recent years, various methods have been proposed to overcome this problem, unfortunately with limited success. In this chapter, we describe in detail a protocol for hemapheresis with an immunoadsorption matrix to remove specifically anti-AAV antibodies in an in vivo rat model. Furthermore, this chapter describes in detail the methods to determine the efficiency of hemapheresis and immunoadsorption.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11338470 | PMC |
| http://dx.doi.org/10.1007/978-1-0716-2707-5_18 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Therapeutic Apheresis in Pediatric Renal Transplant Recipients.
Exp Clin Transplant
October 2024
From the Urology and Nephrology Research Center, Shahidbeheshti of Medical Sciences, Tehran, Iran.
Clear guidelines for therapeutic apheresis in children after renal transplant do not exist. This article reviews the current experiences with therapeutic apheresis in pediatric transplant recipients. The ideal characteristics of removable substances should have all of the following criteria for an effective therapeutic apheresis: large molecular weight (>15 kDa), prolonged half-life, rapid elimination from the plasma, high intravascular distribution, and low turnover rate.
View Article and Find Full Text PDFManagement and long-term outcome of recurrent idiopathic FSGS in pediatric kidney transplant recipients.
Sci Rep
October 2024
Pediatric Nephrology Institute, Ruth Children's Hospital, Rambam Health Care Campus, Haifa, Israel.
Article Synopsis
- Focal segmental glomerulosclerosis (FSGS) is a significant cause of kidney failure in children, predominantly presenting as idiopathic cases which often recur after transplantation.
- Early treatment involving plasmapheresis and rituximab was evaluated in a study of pediatric kidney transplant recipients, showing that 75% experienced recurrence of FSGS post-transplant.
- The results demonstrated that plasmapheresis led to remission in all patients, and rituximab helped further sustain remission and reduce the need for plasmapheresis, with minimal adverse effects noted.
A short-term functional recovery comparison of therapeutic plasma exchange and immunoadsorption in severe acute neuroimmune diseases.
Int Immunopharmacol
October 2024
Department of Nephrology, the Second Xiangya Hospital, Central South University, Changsha, Hunan, China; Hunan Key Laboratory of Kidney Disease and Blood Purification, Changsha, Hunan, China. Electronic address:
Objective: To compare the differential impact of recombinant protein A immunoadsorption (PAIA) or therapeutic plasma exchange (TPE) on neurological functional improvement and quality of life in patients afflicted with severe acute neuroimmune diseases, including Guillain-Barré syndrome (GBS), myasthenia gravis (MG), neuromyelitis optica spectrum disorder (NMOSD), and anti-NMDA receptor encephalitis (NMDARE).
Methods: The retrospective study included 29 patients with moderate to severe disability (modified Rankin scale, mRS≥3) due to acute neuroimmune diseases at the second Xiangya hospital from January 2021 to January 2023. The clinical efficacy of PAIA and TPE in improving neurological function (ΔmRS≥1) and the difference in favorable functional outcomes (mRS 0-2) at three months were evaluated.
Efficacy and safety of apheresis therapy in AQP4 antibody-positive NMOSD attack: A propensity score-matched cohort study.
CNS Neurosci Ther
May 2024
Center for Neurology, Beijing Tiantan Hospital, Capital Medical University, Beijing, China.
Objective: Plasma exchange (PE) and immunoadsorption (IA) are recognized as effective ways to treat attacks in AQP4 antibody-positive NMOSD, but high-quality evidence was lacking. To evaluate the efficacy and safety of PE/IA plus intravenous methylprednisolone (IVMP) in NMOSD attacks using propensity scores to match IVMP as control.
Methods: Patients were from a prospective observational cohort study.
Apheresis treatment in autoimmune neurological diseases: Predictors of good clinical outcome and success of follow-up therapy with B-cell depletion.
J Neurol Sci
June 2024
Department of Neurology, Saarland University Medical Center, 66421 Homburg, Germany. Electronic address:
Objective: Apheresis treatment (AT) is an established standard of treatment in various neurological autoimmune diseases. Since not all patients equally benefit from AT, we saw the need to investigate the effect of different clinical, paraclinical and technical-apparative factors on the clinical outcome. Additionally, we wanted to find out whether patients who improved due to AT continue to be clinically stable under B-cell depletion (BCD).
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!