What are the specific limitations of conventional therapy for genetic diseases?5 answersConventional therapy for genetic diseases has several limitations. One major drawback is the risk of insertional mutagenesis and immunogenicity associated with adeno-associated virus (AAV) vectors. Another limitation is the efficacy limitations of the AAV system. Additionally, the use of vectors derived from viruses, although disarmed, still present risks to patients, such as recombination. Furthermore, gene therapy is a complex process that requires precise gene insertion, tight control of gene expression, and management of toxicity, vectors, and immune responses. These challenges need to be addressed specifically for each disease, making gene therapy a formidable task. Overall, the limitations of conventional therapy for genetic diseases include the risks associated with AAV vectors, the complexity of gene therapy, and the need for disease-specific solutions.
What are the limitations of current HSV treatments?4 answersCurrent HSV treatments, such as acyclovir, have limitations that hinder their effectiveness. Resistance and toxicity are major concerns with nucleoside analogs, which are commonly used antiviral drugs. The emergence of drug-resistant pathogens is a challenge, particularly among immunocompromised patients, and treatment options for acyclovir-resistant HSV are limited. Additionally, antiviral drugs like foscarnet and cidofovir, which are used as second-line treatments, can be associated with significant toxicities and are only available in intravenous formulations. Furthermore, antiviral treatments may not eradicate viral latent infections, and the prolonged use of these drugs can lead to the development of resistance. The absence of licensed prophylactic and therapeutic vaccines against HSV also limits treatment options. These limitations highlight the need for alternative therapies and the development of new antiviral compounds to improve the management of HSV infections.
What are the best medicines used in axial spondylarthritis remission?5 answersNonsteroidal anti-inflammatory drugs (NSAIDs) are considered a first-line therapy in patients with axial spondyloarthritis (axSpA) for achieving remission. NSAIDs reduce pain and stiffness effectively in most patients, are able to reduce systemic and local inflammation, and can inhibit progression of structural damage in the spine. However, effective control of symptoms and retardation of radiographic progression often require continuous and long-term treatment, which raises safety concerns. The recent introduction of specific Cox-2 inhibitors, with a lower risk of severe gastrointestinal adverse events, may modify the discontinuous intake of NSAIDs. In addition to NSAIDs, TNF-α antagonists have shown short-term and long-term efficacy in achieving remission in axial spondyloarthritis, with control of pain, extra-articular manifestations, and spinal inflammation as evidenced by MRI. Therefore, a combination of NSAIDs and TNF-α antagonists may be the best approach for achieving remission in axial spondyloarthritis.
Lipid and amino acid targets as therapy option for osteoarthritis?5 answersLipid and amino acid targets are being explored as potential therapy options for osteoarthritis (OA). In OA, the progressive changes in joint tissues are dependent on active cell-mediated processes. Recent research has focused on the underlying mechanisms involving biochemical cross talk among the cartilage, synovium, bone, and other joint tissues. Fatty acids and derivatives have been found to play a role in OA, as evidenced by their presence in serum and synovial fluid and their association with clinical characteristics. Intervention studies in humans or mouse models of disease have also provided evidence for the role of lipids in OA. Targeted therapies for OA, including those targeting lipids and amino acids, have shown promise in providing pain relief and potentially modifying the disease. Further research is needed to fully understand the role of lipids and amino acids in OA and to validate their potential as therapeutic targets.
More research is needed to develop lipid and amino acid targets as therapy option for osteoarthritis?5 answersMore research is needed to develop lipid and amino acid targets as therapy options for osteoarthritis. Several studies have identified biomarkers for osteoarthritis, including biochemical biomarkers such as collagen degradation products, pro- or anti-inflammatory cytokines, micro RNAs, long non-coding RNAs, and circular RNAs. Abnormal lipid metabolism has been observed in osteoarthritis, and the roles of fatty acids and oxylipins in joint degradation and protection have been studied extensively. Additionally, the potential therapeutic effects of natural dietary supplements, such as olive oil, olive leaf extract, curcumin, sanguinarine, vitamin D, and carnosic acid, have been investigated. However, there is still a lack of effective treatments for joint destruction in osteoarthritis patients, and further research is needed to explore the associations between hyperuricemia, gout, urate lowering therapy, and osteoarthritis. Therefore, more research is necessary to better understand the role of lipids and amino acids in osteoarthritis and develop targeted therapies.
What are the limitations of current treatment options for sickle cell disease (SCD)?2 answersCurrent treatment options for sickle cell disease (SCD) have limitations. Hydroxyurea remains the main treatment option, but it is not widely available in low-income countries. Erythrocyte transfusion has limited evidence-based indications. Hematopoietic stem cell transplant is a curative option, but suitable donors are scarce. The development of new drugs and treatment procedures for SCD has historically been underfunded. However, targeted therapies such as L-glutamine and crizanlizumab have shown promise in improving outcomes. Gene therapy and stem cell transplantation are potential curative approaches, but more research and clinical trials are needed. Premature mortality persists in youth and young adults with SCD, and adults with SCD face increased disease burden and organ damage. These limitations highlight the need for more accessible and effective treatment options for SCD.