Overview of Gene and Genome
Therapies 2026 Expert Verified | Ace the
Test
Gene therapy - 🧠ANSWER ✔✔Aims to replace an entire abnormal gene,
potentially offering a permanent disease state change.
Genome therapy - 🧠ANSWER ✔✔Focuses on altering specific gene parts
to correct function, designed for lasting disruption of pathogenic mutations.
Similarities between gene and genome therapies - 🧠ANSWER ✔✔Both
seek to modify genes for therapeutic benefit.
Differences between gene and genome therapies - 🧠ANSWER ✔✔Gene
therapy replaces whole genes; genome therapy edits specific sequences.
, Potential of Induced pluripotent stem cells (iPSCs) - 🧠ANSWER ✔✔Can be
combined with gene/genome therapies, allowing for the creation of patient-
specific corrected cells.
Why is targeting the right cell important? - 🧠ANSWER ✔✔Selecting the
right target cell (germline or somatic) determines the duration and
heritability of the effects.
Alterations to germline cells - 🧠ANSWER ✔✔Alterations are long-term and
passed to future offspring.
Alteration to Somatic Cells - 🧠ANSWER ✔✔Effects are restricted to the
treated patient.
Considerations - 🧠ANSWER ✔✔Immunogenicity, delivery efficiency, and
off-target effects must be addressed.
Recombinant Viral Particles - 🧠ANSWER ✔✔Modified viruses, like
lentivirus and adenovirus, are commonly used for delivering gene editing
tools.
Nanotechnology - 🧠ANSWER ✔✔Engineered nanoparticles offer tunable
size and surface properties for efficient delivery.
Therapies 2026 Expert Verified | Ace the
Test
Gene therapy - 🧠ANSWER ✔✔Aims to replace an entire abnormal gene,
potentially offering a permanent disease state change.
Genome therapy - 🧠ANSWER ✔✔Focuses on altering specific gene parts
to correct function, designed for lasting disruption of pathogenic mutations.
Similarities between gene and genome therapies - 🧠ANSWER ✔✔Both
seek to modify genes for therapeutic benefit.
Differences between gene and genome therapies - 🧠ANSWER ✔✔Gene
therapy replaces whole genes; genome therapy edits specific sequences.
, Potential of Induced pluripotent stem cells (iPSCs) - 🧠ANSWER ✔✔Can be
combined with gene/genome therapies, allowing for the creation of patient-
specific corrected cells.
Why is targeting the right cell important? - 🧠ANSWER ✔✔Selecting the
right target cell (germline or somatic) determines the duration and
heritability of the effects.
Alterations to germline cells - 🧠ANSWER ✔✔Alterations are long-term and
passed to future offspring.
Alteration to Somatic Cells - 🧠ANSWER ✔✔Effects are restricted to the
treated patient.
Considerations - 🧠ANSWER ✔✔Immunogenicity, delivery efficiency, and
off-target effects must be addressed.
Recombinant Viral Particles - 🧠ANSWER ✔✔Modified viruses, like
lentivirus and adenovirus, are commonly used for delivering gene editing
tools.
Nanotechnology - 🧠ANSWER ✔✔Engineered nanoparticles offer tunable
size and surface properties for efficient delivery.
